0000950170-24-121679 | 10-Q | XBRL Viewer | Ultragenyx Pharmaceutical INC

Cash, Cash Equivalents and Restricted Cash

Basis of Presentation

The accompanying unaudited Condensed Consolidated Financial Statements include the accounts of the Company and its wholly-owned subsidiaries. The Company consolidates any variable interest entity, or VIE, for which it is the primary beneficiary.

The financial statements have been prepared in accordance with U.S. generally accepted accounting principles, or GAAP, for interim financial information and in accordance with the instructions to Form 10-Q and Rule 10-01 of Regulation S-X. Accordingly,

they do not include all of the information and footnotes required by GAAP for complete financial statements. The unaudited interim Condensed Consolidated Financial Statements have been prepared on the same basis as the annual financial statements. In the opinion of management, the accompanying unaudited Condensed Consolidated Financial Statements reflect all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation. All intercompany accounts and transactions have been eliminated. These financial statements should be read in conjunction with the audited financial statements and notes thereto for the preceding fiscal year contained in the Company’s Annual Report on Form 10-K filed on February 21, 2024, or Annual Report, with the United States Securities and Exchange Commission, or the SEC.

The results of operations for the three and nine months ended September 30, 2024 are not necessarily indicative of the results to be expected for the year ending December 31, 2024. The Condensed Consolidated Balance Sheet as of December 31, 2023 has been derived from audited financial statements at that date, but does not include all of the information required by GAAP for complete financial statements.

Use of Estimates

The accompanying Condensed Consolidated Financial Statements have been prepared in accordance with GAAP. The preparation of the Condensed Consolidated Financial Statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent liabilities and the reported amounts of expenses in the Condensed Consolidated Financial Statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical trial accruals, fair value of assets and liabilities, income taxes, stock-based compensation, revenue recognition, and the liabilities for sales of future royalties. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates.

Restricted cash primarily consists of money market accounts used as collateral for the Company’s obligations under its facility leases and to guarantee the fulfillment of certain sales orders to certain government-sponsored customers. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statement of Cash Flows (in thousands):


	September 30,
	2024		2023
Cash and cash equivalents	$	150,619	$	72,575
Restricted cash included in other current assets		6,176		1,976
Restricted cash included in other non-current assets		3,419		4,056
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows	$	160,214	$	78,607

Credit Losses

The Company is exposed to credit losses primarily through receivables from customers and collaborators and through its available-for-sale debt securities. For trade receivables and other instruments, the Company uses a forward-looking expected loss model that generally results in the earlier recognition of allowances for losses. For available-for-sale debt securities with unrealized losses, the losses are recognized as allowances rather than as reductions in the amortized cost of the securities.

The Company’s expected loss allowance methodology for the receivables is developed using historical collection experience, current and future economic market conditions, a review of the current aging status and financial condition of the entities. Specific allowance amounts are established to record the appropriate allowance for customers that have a higher probability of default. Balances are written off when determined to be uncollectible. The Company’s expected loss allowance methodology for the debt securities is developed by reviewing the extent of the unrealized loss, the size, term, geographical location, and industry of the issuer, the issuers’ credit ratings and any changes in those ratings, as well as reviewing current and future economic market conditions and the issuers’ current status and financial condition. There were no material credit losses recorded for receivables and available-for-sale debt securities which were attributable to credit risk for the three and nine months ended September 30, 2024 and 2023.

Revenue Recognition

Product Sales

The Company sells its approved products through a limited number of distributors. Under Accounting Standards Codification, or ASC, 606, Revenue from Contracts with Customers, revenue from product sales is recognized at the point in time when control is transferred to these distributors. The Company also recognizes revenue from sales of certain products on a “named patient” basis, which are allowed in certain countries prior to the commercial approval of the product. Prior to recognizing revenue, the Company makes estimates of the transaction price, including any variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Product sales are recorded net of estimated government-mandated rebates and chargebacks, estimated product returns, and other deductions.

Provisions for returns and other adjustments are provided for in the period the related revenue is recorded, as estimated by management. These reserves are based on estimates of the amounts earned or to be claimed on the related sales and are reviewed periodically and adjusted as necessary. The Company’s estimates of government mandated rebates, chargebacks, estimated product returns, and other deductions depends on the identification of key customer contract terms and conditions, as well as estimates of sales volumes to different classes of payors. If actual results vary, the Company may need to adjust these estimates, which could have a material effect on earnings in the period of the adjustment.

Collaboration, License, and Royalty Revenue

The Company has certain license and collaboration agreements that are within the scope of ASC, 808, Collaborative Agreements, which provides guidance on the presentation and disclosure of collaborative arrangements. Generally, the classification of the transactions under the collaborative arrangements is determined based on the nature of contractual terms of the arrangement, along with the nature of the operations of the participants. The Company records its share of collaboration revenue, net of transfer pricing related to net sales in the period in which such sales occur, if the Company is considered as an agent in the arrangement. The Company is considered an agent when the collaboration partner controls the product before transfer to the customers and has the ability to direct the use of and obtain substantially all of the remaining benefits from the product. Funding received related to research and development services and commercialization costs is generally classified as a reduction of research and development expenses and selling, general and administrative expenses, respectively, in the Condensed Consolidated Statements of Operations, because the provision of such services for collaborative partners are not considered to be part of the Company’s ongoing major or central operations.

The Company utilizes certain information from its collaboration partners to record collaboration revenue, including revenue from the sale of the product, associated reserves on revenue, and costs incurred for development and sales activities. For the periods covered in the financial statements presented, there have been no material changes to prior period estimates of revenues and expenses. The Company also records royalty revenues under certain of the Company’s license or collaboration agreements in exchange for licensing of intellectual property.

The Company sold the right to receive certain future royalty payments from net sales of Crysvita in certain territories to RPI Finance Trust, or RPI, an affiliate of Royalty Pharma, and to OCM LS23 Holdings LP, an investment vehicle for Ontario Municipal Employees Retirement System, or OMERS, as further described in “Note 9. Liabilities for Sales of Future Royalties.” The Company records the royalty revenue from the net sales of Crysvita in the applicable territories on a prospective basis as non-cash royalty revenue in the Condensed Consolidated Statements of Operations over the term of the applicable arrangement.

The terms of the Company’s collaboration and license agreements may contain multiple performance obligations, which may include licenses and research and development activities. The Company evaluates these agreements under ASC 606 to determine the distinct performance obligations. The Company analogizes to ASC 606 for the accounting for distinct performance obligations for which there is a customer relationship. Prior to recognizing revenue, the Company makes estimates of the transaction price, including variable consideration that is subject to a constraint. Amounts of variable consideration are included in the transaction price to the extent that it is probable that a significant reversal in the amount of cumulative revenue recognized will not occur and when the uncertainty associated with the variable consideration is subsequently resolved. Total consideration may include nonrefundable upfront license fees, payments for research and development activities, reimbursement of certain third-party costs, payments based upon the achievement of specified milestones, and royalty payments based on product sales derived from the collaboration.

If there are multiple distinct performance obligations, the Company allocates the transaction price to each distinct performance obligation based on its relative standalone selling price. The standalone selling price is generally determined based on the prices charged to customers or using expected cost-plus margin. The Company estimates the efforts needed to complete the performance obligations and recognizes revenue by measuring the progress towards complete satisfaction of the performance obligations using input measures.

Disclosure of accounting policy for credit loss on financial instrument measured at amortized cost basis, net investment in lease, off-balance sheet credit exposure, and available-for-sale debt security. Includes, but is not limited to, methodology used to estimate allowance for credit loss, how writeoff of uncollectible amount is recognized, and determination of past due status and nonaccrual status.

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Liabilities for Sales of Future Royalties - Additional Information (Details) - USD ($) $ in Millions	1 Months Ended		9 Months Ended	58 Months Ended
	Jul. 31, 2022	Dec. 31, 2019	Sep. 30, 2024	Sep. 30, 2024
OMERS
Liability For Sale Of Future Royalties [Line Items]
Profit loss sharing percentage on net sales	30.00%
Effective annual interest rate			7.60%	7.60%
Royalty Purchase Agreement \| RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Proceeds from sale of future royalties		$ 320.0
Royalty payment termination description			The agreement with RPI will automatically terminate, and the payment of royalties to RPI will cease, in the event aggregate royalty payments received by RPI are equal to or greater than $608.0 million prior to December 31, 2030, or in the event aggregate royalty payments received by RPI are less than $608.0 million prior to December 31, 2030, or when aggregate royalty payments received by RPI are equal to $800.0 million.
Royalties transaction costs net		5.8
Proceeds from the sale of future royalties, net		$ 314.2
Effective annual interest rate			6.20%	6.20%
Non-cash collaboration royalty revenue				$ 91.8
Royalty Purchase Agreement \| OMERS
Liability For Sale Of Future Royalties [Line Items]
Proceeds from sale of future royalties	$ 500.0
Royalty payment termination description	In July 2022, the Company entered into a Royalty Purchase Agreement with OMERS. Pursuant to the agreement, OMERS paid $500.0 million to the Company in consideration for the right to receive 30% of the future royalty payments due to the Company from KKC based on net sales of Crysvita in the U.S. and Canada under the terms of the KKC Collaboration Agreement. The calculation of royalty payments to OMERS is based on net sales of Crysvita beginning in April 2023 and will expire upon the earlier of the date on which aggregate payments received by OMERS equals $725.0 million or the date the final royalty payment is made to the Company under the KKC Collaboration Agreement.
Royalty agreement termination threshold amount	$ 725.0
Payment from sale of future royalties	500.0
Royalties transaction costs net	9.1
Proceeds from the sale of future royalties, net	$ 491.0
Non-cash collaboration royalty revenue				97.6
Royalty Purchase Agreement \| Minimum \| RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Royalty agreement termination threshold amount			$ 608.0	608.0
Royalty Purchase Agreement \| Maximum \| RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Royalty agreement termination threshold amount			$ 800.0	$ 800.0

Equity Transactions - Summary of Pre- Funded Warrants Activity (Details) - Pre Funded Warrant - shares	9 Months Ended	12 Months Ended
	Sep. 30, 2024	Dec. 31, 2023
Stockholders Equity [Line Items]
Beginning Balance	1,666,722	0
Issuance of pre-funded warrants	1,538,501	1,666,722
Ending Balance	3,205,223	1,666,722

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Unaudited) - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-in Capital	Accumulated Other Comprehensive Income (Loss)	Accumulated Deficit	Treasury Stock	Deferred Compensation Obligation
Beginning balance at Dec. 31, 2022	$ 352,494	$ 70	$ 3,140,019	$ (6,573)	$ (2,781,022)
Beginning balance, shares at Dec. 31, 2022		70,197,297
Issuance of common stock in connection with at-the-market offering, net of issuance costs	53,299	$ 1	53,298
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares		1,175,584
Stock-based compensation	100,780		100,780
Issuance of common stock under equity plan awards, net of tax	4,261	$ 1	4,260
Issuance of common stock under equity plan awards, net of tax, shares		801,426
Deferred compensation						$ (381)	$ 381
Other comprehensive income	4,329			4,329
Net Income (Loss)	(483,449)				(483,449)
Ending balance at Sep. 30, 2023	31,714	$ 72	3,298,357	(2,244)	(3,264,471)	(381)	381
Ending balance, shares at Sep. 30, 2023		72,174,307
Beginning balance at Jun. 30, 2023	129,030	$ 71	3,236,879	(3,098)	(3,104,822)	(381)	381
Beginning balance, shares at Jun. 30, 2023		71,465,424
Issuance of common stock in connection with at-the-market offering, net of issuance costs	24,792	$ 1	24,791
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares		611,282
Stock-based compensation	36,245		36,245
Issuance of common stock under equity plan awards, net of tax	442		442
Issuance of common stock under equity plan awards, net of tax, shares		97,601
Other comprehensive income	854			854
Net Income (Loss)	(159,649)				(159,649)
Ending balance at Sep. 30, 2023	31,714	$ 72	3,298,357	(2,244)	(3,264,471)	(381)	381
Ending balance, shares at Sep. 30, 2023		72,174,307
Beginning balance at Dec. 31, 2023	$ 275,414	$ 82	3,662,346	647	(3,387,661)	(432)	432
Beginning balance, shares at Dec. 31, 2023	82,315,590	82,315,590
Issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net	$ 380,983	$ 9	380,974
Issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net, shares		8,782,051
Stock-based compensation	117,907		117,907
Issuance of common stock under equity plan awards, net of tax	8,072	$ 1	8,071
Issuance of common stock under equity plan awards, net of tax, shares		1,206,676
Deferred compensation						(3,123)	3,123
Other comprehensive income	249			249
Net Income (Loss)	(435,798)				(435,798)
Ending balance at Sep. 30, 2024	$ 346,827	$ 92	4,169,298	896	(3,823,459)	(3,555)	3,555
Ending balance, shares at Sep. 30, 2024	92,304,317	92,304,317
Beginning balance at Jun. 30, 2024	$ 432,418	$ 92	4,123,364	(1,095)	(3,689,943)	(3,395)	3,395
Beginning balance, shares at Jun. 30, 2024		92,135,384
Issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net	117		117
Stock-based compensation	41,760		41,760
Issuance of common stock under equity plan awards, net of tax	4,057		4,057
Issuance of common stock under equity plan awards, net of tax, shares		168,933
Deferred compensation						(160)	160
Other comprehensive income	1,991			1,991
Net Income (Loss)	(133,516)				(133,516)
Ending balance at Sep. 30, 2024	$ 346,827	$ 92	$ 4,169,298	$ 896	$ (3,823,459)	$ (3,555)	$ 3,555
Ending balance, shares at Sep. 30, 2024	92,304,317	92,304,317

Equity Transactions

9 Months Ended

Sep. 30, 2024

Equity [Abstract]

Equity Transactions

12. Equity Transactions

At-the-Market Offerings

In February 2024, the Company entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds up to $350.0 million, from time to time, in at-the-market, or ATM, offerings through Cowen. No shares were sold under this agreement during the three and nine months ended September 30, 2024.

Underwritten Public Offering

In October 2023, the Company completed an underwritten public offering in which 9,833,334 shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1,500,000 shares, at a public offering price of $30.00 per share. In connection with the offering, the Company sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1,666,722 shares of common stock at a purchase price of $29.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $0.001 exercise price per share of each pre-funded warrant. The total proceeds that the Company received from the offering were $326.5 million, net of underwriting discounts and commissions.

In June 2024, the Company completed an underwritten public offering in which 8,782,051 shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1,346,153 shares, at a public offering price of $39.00 per share. In connection with the offering, the Company sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1,538,501 shares of common stock at a purchase price of $38.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $0.001 exercise price per share of each pre-funded warrant. The total proceeds that the Company received from the offering were $381.0 million, net of underwriting discounts and commissions.

The pre-funded warrants were classified as a component of permanent equity in the Company's Condensed Consolidated Balance Sheets as they are freestanding financial instruments that are immediately exercisable, do not embody an obligation for the Company to repurchase its own shares and permit the holders to receive a fixed number of shares of common stock upon exercise. All of the shares underlying the pre-funded warrants have been included in the weighted-average number of shares of common stock used to calculate net loss per share, basic and diluted, attributable to common stockholders because the shares may be issued for little or no consideration, are fully vested, and are exercisable after the original issuance date of the pre-funded warrants. As of September 30, 2024, none of the pre-funded warrants had been exercised.

The table below summarizes the pre-funded warrants activity:


	Pre-funded warrants
As of December 31, 2022		—
Issuance of pre-funded warrants		1,666,722
As of December 31, 2023		1,666,722
Issuance of pre-funded warrants		1,538,501
As of September 30, 2024		3,205,223

The entire disclosure for equity.

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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-16

Reference 17: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 18
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Section 50
-Paragraph 18
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

Reference 19: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Section 50
-Paragraph 18
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

Summary of Pre- Funded Warrants Activity

Liabilities for Sales of Future Royalties - Schedule of Activity Within Liability Related to Sale of Future Royalties (Details) - USD ($) $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2024	Dec. 31, 2023
Liability For Sale Of Future Royalties [Line Items]
Liability for sale of future royalties at beginning of year	$ 891,567	$ 875,439
Royalty revenue	(60,669)	(59,307)
Non-cash interest expense	47,519	75,435
Liability for sale of future royalties at end of year	878,417	891,567
RPI Finance Trust (RPI)
Liability For Sale Of Future Royalties [Line Items]
Liability for sale of future royalties at beginning of year	376,641	365,189
Royalty revenue	(18,376)	(20,783)
Non-cash interest expense	17,779	32,235
Liability for sale of future royalties at end of year	376,044	376,641
OMERS
Liability For Sale Of Future Royalties [Line Items]
Liability for sale of future royalties at beginning of year	514,926	510,250
Royalty revenue	(42,293)	(38,524)
Non-cash interest expense	29,740	43,200
Liability for sale of future royalties at end of year	$ 502,373	$ 514,926

Equity Transactions (Tables)

9 Months Ended

Sep. 30, 2024

Equity [Abstract]

The table below summarizes the pre-funded warrants activity:


	Pre-funded warrants
As of December 31, 2022		—
Issuance of pre-funded warrants		1,666,722
As of December 31, 2023		1,666,722
Issuance of pre-funded warrants		1,538,501
As of September 30, 2024		3,205,223

Liabilities for Sales of Future Royalties

9 Months Ended

Sep. 30, 2024

Deferred Revenue Disclosure [Abstract]

Liabilities for Sales of Future Royalties

9. Liabilities for Sales of Future Royalties

In December 2019, the Company entered into a Royalty Purchase Agreement with RPI. Pursuant to the agreement, RPI paid $320.0 million to the Company in consideration for the right to receive royalty payments effective January 1, 2020, arising from the net sales of Crysvita in the EU, the U.K., and Switzerland under the terms of the Company’s Collaboration and License Agreement with KKC dated August 29, 2013, as amended, or the KKC Collaboration Agreement. The agreement with RPI will automatically terminate, and the payment of royalties to RPI will cease, in the event aggregate royalty payments received by RPI are equal to or greater than $608.0 million prior to December 31, 2030, or in the event aggregate royalty payments received by RPI are less than $608.0 million prior to December 31, 2030, or when aggregate royalty payments received by RPI are equal to $800.0 million.

In July 2022, the Company entered into a Royalty Purchase Agreement with OMERS. Pursuant to the agreement, OMERS paid $500.0 million to the Company in consideration for the right to receive 30% of the future royalty payments due to the Company from KKC based on net sales of Crysvita in the U.S. and Canada under the terms of the KKC Collaboration Agreement. The calculation of royalty payments to OMERS is based on net sales of Crysvita beginning in April 2023 and will expire upon the earlier of the date on which aggregate payments received by OMERS equals $725.0 million or the date the final royalty payment is made to the Company under the KKC Collaboration Agreement.

Proceeds from these transactions were recorded as liabilities for sales of future royalties on the Condensed Consolidated Balance Sheets. Upon inception of the respective arrangements, the Company recorded $320.0 million and $500.0 million, net of transaction costs of $5.8 million and $9.1 million for RPI and OMERS, respectively, using the effective interest method over the estimated life of the applicable arrangement. In order to determine the amortization of the liabilities, the Company is required to estimate the total amount of future royalty payments to be received by the Company and paid to RPI and OMERS, subject to the capped amount, over the life of the arrangements. The excess of future estimated royalty payments (subject to the capped amount to RPI and OMERS), over the $314.2 million and $491.0 million, respectively, of net proceeds, is recorded as non-cash interest expense over the life of the arrangements. Consequently, the Company estimates an imputed interest on the unamortized portion of the liabilities and records interest expense relating to the transactions. The Company records the royalty revenue arising from the net sales of Crysvita in the applicable territories as royalty revenue in the Condensed Consolidated Statements of Operations over the term of the arrangements. Royalties earned under the RPI and OMERS arrangements from inception to September 30, 2024 have been $91.8 million and $97.6 million, respectively.

The Company periodically assesses the expected royalty payments using a combination of historical results, internal projections and forecasts from external sources. To the extent such payments are greater or less than the Company’s initial estimates or the timing of such payments is materially different than its original estimates, the Company will prospectively adjust the amortization of the liabilities and the effective interest rate. The Company’s effective annual interest rates were 6.2% and 7.6%, for RPI and OMERS, respectively, as of September 30, 2024.

There are a number of factors that could materially affect the amount and timing of royalty payments from KKC in the applicable territories, most of which are not within the Company’s control. Such factors include, but are not limited to, the success of KKC’s sales and promotion of Crysvita, changing standards of care, macroeconomic and inflationary pressures, the introduction of competing products, pricing for reimbursement in various territories, manufacturing or other delays, intellectual property matters, adverse events that result in governmental health authority imposed restrictions on the use of Crysvita, significant changes in foreign exchange rates as the royalty payments are made in U.S. dollars, or USD, while significant portions of the underlying sales of Crysvita are made in currencies other than USD, and other events or circumstances that could result in reduced royalty payments from sales of Crysvita, all of which would result in a reduction of royalty revenue and the non-cash interest expense over the life of

the arrangement. Conversely, if sales of Crysvita in the relevant territories are more than expected, the royalty revenue and the non-cash interest expense recorded by the Company would be greater over the term of the arrangements.

The following table shows the activity within the liability account (in thousands):


	Liabilities for Sales of Future Royalties
	RPI			OMERS			Total
December 31, 2022	$	365,189		$	510,250		$	875,439
Royalty revenue		(20,783	)		(38,524	)		(59,307	)
Non-cash interest expense		32,235			43,200			75,435
December 31, 2023		376,641			514,926			891,567
Royalty revenue		(18,376	)		(42,293	)		(60,669	)
Non-cash interest expense		17,779			29,740			47,519
September 30, 2024	$	376,044		$	502,373		$	878,417

Balance Sheet Components - Additional Information (Details)	9 Months Ended
	Sep. 30, 2024
Maximum
Schedule Of Available For Sale Securities [Line Items]
Available-for-sale securities remaining contractual maturities	3 years

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
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Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 320
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-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Revenue from Contract with Customer [Abstract]

Revenue

9 Months Ended

Sep. 30, 2024

Revenue

5. Revenue

The following table disaggregates total revenues from external customers by product sales, royalty revenue, and collaboration and license revenue (in thousands):


	Three Months Ended				Nine Months Ended
	September 30,				September 30,
	2024		2023		2024		2023
Product sales:
Crysvita	$	35,604	$	19,200	$	112,294	$	57,318
Dojolvi		21,374		16,553		57,091		47,347
Mepsevii		9,616		5,633		22,372		22,552
Evkeeza		10,657		963		21,788		1,540
Total product sales		77,251		42,349		213,545		128,757
Crysvita royalty revenue		62,243		55,703		181,808		106,916
Collaboration and license revenue:
Crysvita collaboration revenue in Profit- Share Territory		—		—		—		69,705
Daiichi Sankyo		—		—		—		1,479
Total collaboration and license revenue		—		—		—		71,184
Total revenues	$	139,494	$	98,052	$	395,353	$	306,857

The following table disaggregates total revenues based on geographic location (in thousands):


	Three Months Ended				Nine Months Ended
	September 30,				September 30,
	2024		2023		2024		2023
North America	$	76,914	$	67,811	$	223,485	$	214,232
Latin America		38,048		17,229		112,155		59,096
Europe, Middle East, and Africa		22,222		13,012		54,352		32,351
Asia-Pacific		2,310		—		5,361		1,178
Total revenues	$	139,494	$	98,052	$	395,353	$	306,857

The Company’s largest accounts receivable balance was from a collaboration partner and accounted for 60% and 53% of the total accounts receivable balance as of September 30, 2024 and December 31, 2023, respectively.

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479806/606-10-50-9

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-URI https://asc.fasb.org/1943274/2147479806/606-10-50-12

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-URI https://asc.fasb.org/1943274/2147479806/606-10-50-12

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-Section 50
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-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479806/606-10-50-12

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479806/606-10-50-12

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
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-Section 50
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-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479806/606-10-50-12

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (b)(2)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147479806/606-10-50-13

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
-Topic 606
-Publisher FASB
-URI https://asc.fasb.org/606/tableOfContent

Share of Collaboration and Royalty Revenue Related to Crysvita

License and Research Agreements - Schedule of Changes in Fair Value of Equity Investment (Details) - USD ($) $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2024	Dec. 31, 2023
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Common stock, Beginning balance	$ 749,239
Common stock, Ending balance	793,719	$ 749,239
Solid and Arcturus \| Common Stock
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Common stock, Beginning balance	3,204	2,807
Common stock, Change in fair value	433	397
Common stock, Ending balance	$ 3,637	$ 3,204

License and Research Agreements (Tables)

9 Months Ended

Sep. 30, 2024

Total Crysvita revenue was as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2024		2023		2024		2023
Revenue in Profit-Share Territory:
Royalty revenue	$	39,189	$	35,160	$	114,402	$	64,221
Non-cash royalty revenue		16,796		15,070		49,030		27,524
Collaboration revenue		—		—		—		69,705
Total revenue in Profit-Share Territory		55,985		50,230		163,432		161,450
Product sales		35,604		19,200		112,294		57,318
Non-cash royalty revenue in European Territories		6,258		5,473		18,376		15,171
Total Crysvita revenue	$	97,847	$	74,903	$	294,102	$	233,939

Schedule of Cost Sharing Payments

These amounts were recognized in the Company’s Condensed Consolidated Statements of Operations in connection with the collaboration agreement with KKC as follows (in thousands):


	Three Months Ended September 30,						Nine Months Ended September 30,
	2024			2023			2024			2023
Research and development	$	(841	)	$	(940	)	$	(2,779	)	$	(4,665	)
Selling, general and administrative		(898	)		(2,060	)		(3,184	)		(15,346	)
Cost of sales		12,044			4,661			37,952			14,189

Solid and Arcturus

Schedule of Changes in Fair Value of Equity Investment

The changes in the fair value of the Company’s equity investment in the common stock of Solid were as follows (in thousands):


	Common stock
As of December 31, 2022	$	2,807
Change in fair value		397
As of December 31, 2023		3,204
Change in fair value		433
As of September 30, 2024	$	3,637

Balance Sheet Components (Tables)

9 Months Ended

Sep. 30, 2024

Investments, Debt and Equity Securities [Abstract]

Summary of Cash Equivalents and Marketable Debt Securities Classified as Available For Sale Securities

The fair values of cash equivalents and marketable debt securities classified as available-for-sale securities consisted of the following (in thousands):


	September 30, 2024
				Gross Unrealized
		Amortized Cost		Gains		Losses			Estimated Fair Value
Money market funds		$	85,707	$	—	$	—		$	85,707
Certificates of deposit and time deposits			14,142		—		—			14,142
Corporate bonds			422,228		2,177		(31	)		424,374
Commercial paper			22,072		—		—			22,072
Asset-backed securities			243		—		—			243
U.S. Government Treasury and agency securities			228,008		927		—			228,935
Total		$	772,400	$	3,104	$	(31	)	$	775,473


	December 31, 2023
				Gross Unrealized
		Amortized Cost		Gains		Losses			Estimated Fair Value
Money market funds		$	162,289	$	—	$	—		$	162,289
Certificates of deposit and time deposits			17,986		—		—			17,986
Corporate bonds			214,792		711		(337	)		215,166
Commercial paper			20,620		—		—			20,620
Asset-backed securities			2,715		—		(3	)		2,712
U.S. Government Treasury and agency securities			316,160		982		(100	)		317,042
Total		$	734,562	$	1,693	$	(440	)	$	735,815

Summary of Inventory

Inventory consists of the following (in thousands):


	September 30,		December 31,
	2024		2023
Work-in-process	$	20,281	$	18,859
Finished goods		23,053		15,110
Total inventory	$	43,334	$	33,969

Summary of Accrued Liabilities

Accrued liabilities consist of the following (in thousands):


	September 30,		December 31,
	2024		2023
Research, clinical study, and manufacturing expenses	$	39,752	$	65,326
Payroll and related expenses		82,924		82,936
Revenue related reserves		32,928		17,029
Other		29,424		31,195
Total accrued liabilities	$	185,028	$	196,486

Tabular disclosure of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-9

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 11: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
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Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
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-Section 50
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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Other Comprehensive Income (Loss), Net of Tax [Abstract]

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Sep. 30, 2024 shares
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	On September 9, 2024, Michael Narachi, a member of the Company’s Board of Directors, adopted a trading arrangement intended to satisfy the affirmative defense of Rule 10b5-1(c), to sell up to 16,250 shares of the Company’s common stock, all of which shares are to be acquired upon the exercise of employee stock options, through June 17, 2025, subject to certain conditions.
Name	Michael Narachi
Title	member of the Company’s Board of Directors
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	September 9, 2024
Expiration Date	June 17, 2025
Arrangement Duration	281 days
Aggregate Available	16,250

Revenue - Summary of Disaggregation of Total Revenues from Customers (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Disaggregation Of Revenue [Line Items]
Total revenues	$ 139,494	$ 98,052	$ 395,353	$ 306,857
Collaboration and license
Disaggregation Of Revenue [Line Items]
Total revenues	0	0	0	71,184
Collaboration and license \| Crysvita
Disaggregation Of Revenue [Line Items]
Total revenues	0	0	0	69,705
Collaboration and license \| Daiichi Sankyo
Disaggregation Of Revenue [Line Items]
Total revenues	0	0	0	1,479
Product Revenue
Disaggregation Of Revenue [Line Items]
Total revenues	77,251	42,349	213,545	128,757
Product Revenue \| Crysvita
Disaggregation Of Revenue [Line Items]
Total revenues	35,604	19,200	112,294	57,318
Product Revenue \| Dojolvi
Disaggregation Of Revenue [Line Items]
Total revenues	21,374	16,553	57,091	47,347
Product Revenue \| Mepsevii
Disaggregation Of Revenue [Line Items]
Total revenues	9,616	5,633	22,372	22,552
Product Revenue \| Evkeeza
Disaggregation Of Revenue [Line Items]
Total revenues	10,657	963	21,788	1,540
Royalty revenue
Disaggregation Of Revenue [Line Items]
Total revenues	62,243	55,703	181,808	106,916
Royalty revenue \| Crysvita
Disaggregation Of Revenue [Line Items]
Total revenues	$ 62,243	$ 55,703	$ 181,808	$ 106,916

Related Party Transaction - Additional Information (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended		9 Months Ended		27 Months Ended
	Jul. 31, 2022	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024
Related Party Transaction [Line Items]
Research and development		$ 170,109	$ 157,245	$ 510,099	$ 487,892
Total Contract Value	$ 1,000
Contribution period	4 years
Non Profit Foundation [Member]
Related Party Transaction [Line Items]
Research and development		$ 0	$ 0	$ 300	$ 300	$ 800

Accumulated Other Comprehensive Income (Tables)

9 Months Ended

Sep. 30, 2024

Schedule of Total Accumulated Other Comprehensive Income

Total accumulated other comprehensive income consisted of the following (in thousands):


	September 30,			December 31,
	2024			2023
Foreign currency translation adjustments	$	(2,177	)	$	(606	)
Unrealized gain on available-for-sale securities		3,073			1,253
Total accumulated other comprehensive income	$	896		$	647

Balance Sheet Components - Summary of Inventory (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Inventory, Net [Abstract]
Work-in-process	$ 20,281	$ 18,859
Finished goods	23,053	15,110
Total inventory	$ 43,334	$ 33,969

Revenue (Tables)

9 Months Ended

Sep. 30, 2024

Revenue from Contract with Customer [Abstract]

Summary of Disaggregation of Total Revenues

The following table disaggregates total revenues from external customers by product sales, royalty revenue, and collaboration and license revenue (in thousands):


	Three Months Ended				Nine Months Ended
	September 30,				September 30,
	2024		2023		2024		2023
Product sales:
Crysvita	$	35,604	$	19,200	$	112,294	$	57,318
Dojolvi		21,374		16,553		57,091		47,347
Mepsevii		9,616		5,633		22,372		22,552
Evkeeza		10,657		963		21,788		1,540
Total product sales		77,251		42,349		213,545		128,757
Crysvita royalty revenue		62,243		55,703		181,808		106,916
Collaboration and license revenue:
Crysvita collaboration revenue in Profit- Share Territory		—		—		—		69,705
Daiichi Sankyo		—		—		—		1,479
Total collaboration and license revenue		—		—		—		71,184
Total revenues	$	139,494	$	98,052	$	395,353	$	306,857

The following table disaggregates total revenues based on geographic location (in thousands):


	Three Months Ended				Nine Months Ended
	September 30,				September 30,
	2024		2023		2024		2023
North America	$	76,914	$	67,811	$	223,485	$	214,232
Latin America		38,048		17,229		112,155		59,096
Europe, Middle East, and Africa		22,222		13,012		54,352		32,351
Asia-Pacific		2,310		—		5,361		1,178
Total revenues	$	139,494	$	98,052	$	395,353	$	306,857

Financial Instruments

9 Months Ended

Sep. 30, 2024

Fair Value Disclosures [Abstract]

Financial Instruments

3. Financial Instruments

Financial assets and liabilities are recorded at fair value. The carrying amount of certain financial instruments, including cash and cash equivalents, accounts receivable, accounts payable and accrued liabilities, approximate fair value due to their relatively short maturities. The carrying amounts of liabilities for the sales of future royalties also approximate their fair value. Assets and liabilities recorded at fair value on a recurring basis in the balance sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or an exit price that would be paid to transfer a liability in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The authoritative guidance on fair value measurements establishes a three-tier fair value hierarchy for disclosure of fair value measurements as follows:

Level 1—Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date;

Level 2—Inputs are observable, unadjusted quoted prices in active markets for similar assets or liabilities, unadjusted quoted prices for identical or similar assets or liabilities in markets that are not active, or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the related assets or liabilities; and

Level 3—Unobservable inputs that are significant to the measurement of the fair value of the assets or liabilities that are supported by little or no market data.

The Company determines the fair value of its equity investment in Solid Biosciences Inc., or Solid, by using the quoted market prices, which are Level 1 fair value measurements.

The following tables set forth the fair value of the Company’s financial assets and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands):


	September 30, 2024
	Level 1		Level 2		Level 3		Total
Financial Assets:
Money market funds	$	85,707	$	—	$	—	$	85,707
Certificates of deposit and time deposits		—		14,142		—		14,142
Corporate bonds		—		424,374		—		424,374
Commercial paper		—		22,072		—		22,072
Asset-backed securities		—		243		—		243
U.S. Government Treasury and agency securities		30,781		198,154		—		228,935
Investment in Solid common stock		3,637		—		—		3,637
Deferred compensation assets		—		14,609		—		14,609
Total financial assets	$	120,125	$	673,594	$	—	$	793,719

Financial Liabilities:
Deferred compensation liabilities	$	—	$	14,902	$	—	$	14,902


	December 31, 2023
	Level 1		Level 2		Level 3		Total
Financial Assets:
Money market funds	$	162,289	$	—	$	—	$	162,289
Certificates of deposit and time deposits		—		17,986		—		17,986
Corporate bonds		—		215,166		—		215,166
Commercial paper		—		20,620		—		20,620
Asset-backed securities		—		2,712		—		2,712
U.S. Government Treasury and agency securities		57,437		259,605		—		317,042
Investment in Solid common stock		3,204		—		—		3,204
Deferred compensation assets		—		10,220		—		10,220
Total financial assets	$	222,930	$	526,309	$	—	$	749,239

Financial Liabilities:
Deferred compensation liabilities	$	—	$	10,365	$	—	$	10,365

Liabilities for Sales of Future Royalties (Tables)

9 Months Ended

Sep. 30, 2024

Deferred Revenue Disclosure [Abstract]

Schedule of Activity Within Liability for Sale of Future Royalties

The following table shows the activity within the liability account (in thousands):


	Liabilities for Sales of Future Royalties
	RPI			OMERS			Total
December 31, 2022	$	365,189		$	510,250		$	875,439
Royalty revenue		(20,783	)		(38,524	)		(59,307	)
Non-cash interest expense		32,235			43,200			75,435
December 31, 2023		376,641			514,926			891,567
Royalty revenue		(18,376	)		(42,293	)		(60,669	)
Non-cash interest expense		17,779			29,740			47,519
September 30, 2024	$	376,044		$	502,373		$	878,417

Investment in Amlogenyx. Inc. - Additional Information (Details) - USD ($) $ in Thousands, shares in Millions	Sep. 30, 2024	Jul. 31, 2024	Dec. 31, 2023
Schedule of Investments [Line Items]
Total assets	$ 1,538,363		$ 1,491,013
Noncontrolling interest	7,000		$ 0
Amlogenyx Inc [Member]
Schedule of Investments [Line Items]
Shares received		9.0
Contributed by affiliated entities		$ 7,000
Total assets	$ 14,100
Amlogenyx Inc [Member] \| Third-Party Investor [Member]
Schedule of Investments [Line Items]
Estimated fair value		$ 7,000
Amlogenyx Inc [Member] \| Series seed preferred stock [Member]
Schedule of Investments [Line Items]
Shares received		1.6

Amount of asset recognized for present right to economic benefit.

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Cost of the investment.

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Fair value of investment in security owned.

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-Subparagraph (SX 210.12-14(Column A)(Footnote 1)(b)(2))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477271/946-320-S99-6

Reference 54: http://www.xbrl.org/2003/role/disclosureRef
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Reference 55: http://www.xbrl.org/2003/role/disclosureRef
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Reference 56: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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Reference 57: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
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Reference 58: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
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-Paragraph 6
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Reference 59: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
-Section S99
-Paragraph 6
-Subparagraph (SX 210.12-14(Column F)(Footnote 4))
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-URI https://asc.fasb.org/1943274/2147477271/946-320-S99-6

Reference 60: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 320
-Name Accounting Standards Codification
-Section S99
-Paragraph 6
-Subparagraph (SX 210.12-14(Column F)(Footnote 5)(a))
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Reference 61: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 320
-Name Accounting Standards Codification
-Section S99
-Paragraph 6
-Subparagraph (SX 210.12-14(Column F)(Footnote 7))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477271/946-320-S99-6

Reference 62: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 320
-Name Accounting Standards Codification
-Section S99
-Paragraph 6
-Subparagraph (SX 210.12-14(Column F)(Footnote 8))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477271/946-320-S99-6

Reference 63: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 320
-Name Accounting Standards Codification
-Section S99
-Paragraph 6
-Subparagraph (SX 210.12-14(Column F)(Footnote 9))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477271/946-320-S99-6

Reference 64: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 320
-Name Accounting Standards Codification
-Section S99
-Paragraph 19
-Subparagraph (1)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477271/946-320-S99-19

Reference 65: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 320
-Name Accounting Standards Codification
-Section S99
-Paragraph 19
-Subparagraph (3)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477271/946-320-S99-19

Carrying amount, attributable to parent and noncontrolling interests, of an entity's issued and outstanding stock which is not included within permanent equity. Temporary equity is a security with redemption features that are outside the control of the issuer, is not classified as an asset or liability in conformity with GAAP, and is not mandatorily redeemable. Includes any type of security that is redeemable at a fixed or determinable price or on a fixed or determinable date or dates, is redeemable at the option of the holder, or has conditions for redemption which are not solely within the control of the issuer. Includes stock with a put option held by an ESOP and stock redeemable by a holder only in the event of a change in control of the issuer.

Related Party Transaction

Equity Transactions - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions	1 Months Ended			3 Months Ended	9 Months Ended	12 Months Ended
	Jun. 30, 2024	Feb. 29, 2024	Oct. 31, 2023	Sep. 30, 2024	Sep. 30, 2024	Dec. 31, 2023
Pre Funded Warrant
Stockholders Equity [Line Items]
Underwritten Public Offering Shares					1,538,501	1,666,722
Number of warrants exercised				0	0
Open Market Sale
Stockholders Equity [Line Items]
Common stock shares sold				0	0
Option to sell common stock aggregate offering price		$ 350.0
Underwritten Public Offering [Member]
Stockholders Equity [Line Items]
Common stock shares sold	8,782,051		9,833,334
Shares purchased by underwriters	1,346,153		1,500,000
Public offering price	$ 39		$ 30
Underwritten Public Offering [Member] \| Pre Funded Warrant
Stockholders Equity [Line Items]
Underwritten Public Offering Shares	1,538,501		1,666,722
Pre-funded warrant price	$ 38.999		$ 29.999
Common stock warrants, exercise price	$ 0.001		$ 0.001
Net proceeds from sale of common stock	$ 381.0		$ 326.5

Related Party Transaction

9 Months Ended

Sep. 30, 2024

13. Related Party Transaction

In July 2022, the Company entered into an agreement with a non-profit foundation in which two members of the Company’s board of directors, including the Company’s Chief Executive Officer, at the time also served as board members of the foundation, whereby an aggregate $1.0 million contribution is being paid to the foundation over a four-year period, beginning in the third quarter of 2022, to support rare disease education and awareness. As a result, the Company recorded nil and $0.3 million as research and development expense for this agreement for the three and nine months ended September 30, 2024, respectively, and nil and $0.3 million for the three and nine months ended September 30, 2023. A total of $0.8 million has been recorded as research and development expense for this agreement to date.

The entire disclosure for related party transactions. Examples of related party transactions include transactions between (a) a parent company and its subsidiary; (b) subsidiaries of a common parent; (c) and entity and its principal owners; and (d) affiliates.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480990/946-20-50-2

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/1943274/2147480990/946-20-50-5

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147480990/946-20-50-6

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-URI https://asc.fasb.org/1943274/2147477968/946-235-50-2

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
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-Paragraph 2
-Subparagraph (e)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147477968/946-235-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
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-Name Accounting Standards Codification
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-Paragraph 1
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483326/850-10-50-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
-Section S99
-Paragraph 1
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
-Section S99
-Paragraph 1
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-Publisher FASB
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-Name Accounting Standards Codification
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-URI https://asc.fasb.org/850/tableOfContent

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
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-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483326/850-10-50-6

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483326/850-10-50-1

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 850
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147483326/850-10-50-1

Preferred stock, par value

CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) (Unaudited) - USD ($)
$ in Thousands

Sep. 30, 2024

Dec. 31, 2023

$ 0.001

Preferred stock, shares authorized

25,000,000

Preferred stock, shares outstanding

Common stock, par value

$ 0.001

Common stock, shares authorized

250,000,000

Common stock, shares outstanding

92,304,317

82,315,590

Treasury stock , shares

68,923

9,559

^[1]

$ 150,619

$ 213,584

Amlogenyx Inc [Member]

$ 14,100

[1]	The Company's Condensed Consolidated Balance Sheet as of September 30, 2024 includes $14.1 million in cash and cash equivalents that can be used only to settle obligations of the consolidated variable interest entity. See Note 6.

License and Research Agreements - Share of Collaboration and Royalty Revenue Related to Crysvita (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	$ 139,494	$ 98,052	$ 395,353	$ 306,857
Royalty revenue
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	62,243	55,703	181,808	106,916
Royalty revenue \| Kyowa Kirin Collaboration \| Profit Share Territory
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	39,189	35,160	114,402	64,221
Non-cash royalty revenue \| Kyowa Kirin Collaboration \| Profit Share Territory
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	16,796	15,070	49,030	27,524
Non-cash royalty revenue \| Kyowa Kirin Collaboration \| Royalty Revenue in European Territories
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	6,258	5,473	18,376	15,171
Collaboration Revenue \| Kyowa Kirin Collaboration \| Profit Share Territory
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	0	0	0	69,705
Collaboration and Royalty \| Kyowa Kirin Collaboration
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	97,847	74,903	294,102	233,939
Collaboration and Royalty \| Kyowa Kirin Collaboration \| Profit Share Territory
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	55,985	50,230	163,432	161,450
Product sales
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	77,251	42,349	213,545	128,757
Product sales \| Kyowa Kirin Collaboration
Disaggregation Of Revenue [Line Items]
Total Crysvita revenue	$ 35,604	$ 19,200	$ 112,294	$ 57,318

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Revenues:
Total revenues	$ 139,494	$ 98,052	$ 395,353	$ 306,857
Operating expenses:
Cost of sales	21,021	10,987	59,834	33,158
Research and development	170,109	157,245	510,099	487,892
Selling, general and administrative	80,351	74,917	239,115	232,966
Total operating expenses	271,481	243,149	809,048	754,016
Loss from operations	(131,987)	(145,097)	(413,695)	(447,159)
Interest income	10,731	5,881	26,956	18,135
Change in fair value of equity investments	678	(1,419)	433	(1,492)
Non-cash interest expense on liabilities for sales of future royalties	(15,712)	(17,665)	(47,519)	(48,676)
Other income (expense)	3,077	(699)	(357)	(2,380)
Loss before income taxes	(133,213)	(158,999)	(434,182)	(481,572)
Provision for income taxes	(303)	(650)	(1,616)	(1,877)
Net loss	$ (133,516)	$ (159,649)	$ (435,798)	$ (483,449)
Net loss per share, basic	$ (1.4)	$ (2.23)	$ (4.91)	$ (6.81)
Net loss per share, diluted	$ (1.4)	$ (2.23)	$ (4.91)	$ (6.81)
Shares used in computing net loss per share, Basic	95,493,996	71,664,493	88,811,157	70,987,801
Shares used in computing net loss per share, Diluted	95,493,996	71,664,493	88,811,157	70,987,801
Product sales
Revenues:
Total revenues	$ 77,251	$ 42,349	$ 213,545	$ 128,757
Royalty revenue
Revenues:
Total revenues	62,243	55,703	181,808	106,916
Collaboration and license
Revenues:
Total revenues	$ 0	$ 0	$ 0	$ 71,184

Investment in Amlogenyx. Inc. - Changes in the Carrying Value of Noncontrolling Interest (Details) $ in Thousands	9 Months Ended
	Sep. 30, 2024 USD ($)
Investments, All Other Investments [Abstract]
As of December 31, 2023	$ 0
Issuance of equity from noncontrolling interest	7,000
As of September 30, 2024	$ 7,000

Share-Based Payment Arrangement [Abstract]

Stock-Based Awards (Tables)

9 Months Ended

Sep. 30, 2024

Summary of Stock-Based Compensation Expense

The table below sets forth the stock-based compensation expense for the periods presented (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2024		2023		2024		2023
Cost of sales	$	428	$	191	$	1,113	$	862
Research and development		22,569		19,113		64,784		56,371
Selling, general and administrative		18,572		15,573		51,969		44,236
Total stock-based compensation expense	$	41,569	$	34,877	$	117,866	$	101,469

Summary of Significant Accounting Policies - Additional Information (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Accounting Policies [Abstract]
Credit losses recorded for receivables	$ 0	$ 0	$ 0	$ 0

License and Research Agreements - Additional Information (Details) - USD ($) $ in Thousands		1 Months Ended					3 Months Ended		9 Months Ended		113 Months Ended
	Jul. 01, 2022	May 31, 2024	Jan. 31, 2024	Jul. 31, 2023	Jan. 31, 2022	Jan. 31, 2021	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2022	Dec. 31, 2023
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Research and development							$ 170,109	$ 157,245	$ 510,099	$ 487,892
Cost of sales							21,021	10,987	59,834	33,158
Regeneron Pharmaceuticals, Inc.
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Upfront payments of collaboration					$ 30,000
Eligible future milestone payments					$ 63,000
Development costs							300	1,900	2,500	6,900
AccruedCollaborationPayables							2,500		2,500			$ 10,600
Regulatory milestone payments achieved		$ 2,500	$ 10,000
Cost of sales							2,700	200	$ 5,400	400
Regeneron Pharmaceuticals, Inc. \| Maximum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Profit loss sharing percentage on net sales					40.00%
Regeneron Pharmaceuticals, Inc. \| Minimum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Profit loss sharing percentage on net sales					20.00%
License Agreement \| Royalty Revenue in European Territory
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Percentage of royalty on net sales receives									10.00%
License and Collaboration Agreement \| Mereo
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Payments made under agreement						$ 50,000
Research and development				$ 9,000
License and Collaboration Agreement \| Maximum \| Mereo
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Future contingent milestone payments							245,000		$ 245,000
Kyowa Kirin Collaboration
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Cost of sales							12,044	$ 4,661	$ 37,952	$ 14,189
Kyowa Kirin Collaboration \| License Agreement
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Profit loss sharing percentage on net sales									30.00%		35.00%
Kyowa Kirin Collaboration \| License Agreement \| Maximum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Tiered double-digit revenue share percentage entitled to receive									30.00%
Kyowa Kirin Collaboration \| License Agreement \| Minimum
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Tiered double-digit revenue share percentage entitled to receive									20.00%
Kyowa Kirin Collaboration \| License Agreement \| Other Current Assets
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
License agreement other receivables							1,800		$ 1,800			1,100
Kyowa Kirin Collaboration \| License Agreement \| Profit Share Revenue
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
License agreement accounts receivable							55,900		55,900			39,200
Kyowa Kirin Collaboration \| License Agreement \| Commercial and Development Activity Reimbursements
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
License agreement accrued liabilities							$ 11,700		$ 11,700			$ 5,300
Kyowa Kirin Collaboration \| License Agreement \| Profit Share Territory
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Percentage of right to receive royalty payments on net sales	30.00%
Remaining profit or loss share percentage on commercializing products									50.00%

Document and Entity Information - shares	9 Months Ended
Document and Entity Information - shares	Sep. 30, 2024	Oct. 31, 2024
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Sep. 30, 2024
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q3
Trading Symbol	RARE
Entity Registrant Name	ULTRAGENYX PHARMACEUTICAL INC.
Entity Central Index Key	0001515673
Entity Current Reporting Status	Yes
Current Fiscal Year End Date	--12-31
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Common Stock, Shares Outstanding		92,344,268
Entity Shell Company	false
Entity File Number	001-36276
Entity Tax Identification Number	27-2546083
Entity Address, Address Line One	60 Leveroni Court
Entity Address, City or Town	Novato
Entity Address, State or Province	CA
Entity Address, Postal Zip Code	94949
City Area Code	415
Local Phone Number	483-8800
Entity Interactive Data Current	Yes
Title of 12(b) Security	Common Stock, $0.001 par value
Security Exchange Name	NASDAQ
Entity Incorporation, State or Country Code	DE
Document Quarterly Report	true
Document Transition Report	false

Net Loss Per Share

9 Months Ended

Sep. 30, 2024

Earnings Per Share [Abstract]

Net Loss Per Share

11. Net Loss Per Share

Basic net loss per share has been computed by dividing the net loss by the weighted-average number of shares of common stock outstanding, pre-funded warrants, and treasury stock for deferred compensation obligations required to be settled in shares of common stock. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of shares of common stock outstanding, pre-funded warrants, and treasury stock for deferred compensation obligations required to be settled in shares of common stock, and potential dilutive securities outstanding during the period.

The following weighted-average outstanding common stock equivalents were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2024		2023		2024		2023
Options to purchase common stock, restricted stock units, and performance stock units		16,667,134		14,585,014		16,235,303		14,117,741
Employee stock purchase plan		65,560		66,735		36,369		37,156
		16,732,694		14,651,749		16,271,672		14,154,897

GeneTx Acquisition

9 Months Ended

Sep. 30, 2024

Business Combinations [Abstract]

GeneTx Acquisition

. GeneTx Acquisition

In August 2019, the Company entered into a Program Agreement and a Unitholder Option Agreement with GeneTx Biotherapeutics LLC, or GeneTx, to collaborate on the development of GeneTx’s GTX-102, an ASO for the treatment of Angelman syndrome. In July 2022, pursuant to the terms of the Unitholder Option Agreement, as amended, the Company exercised the option to acquire GeneTx and entered into a Unit Purchase Agreement, or the Purchase Agreement, pursuant to which the Company purchased all the outstanding units of GeneTx. In accordance with the terms of the Purchase Agreement, the Company paid the option exercise price of $75.0 million and an additional $15.6 million to acquire the outstanding cash of GeneTx, and adjustments for working capital and transaction expenses of $0.6 million, for a total purchase consideration of $91.2 million. The Company may be required to make payments of up to $190.0 million upon the achievement of certain milestones, including up to $30.0 million in milestone payments upon achievement of the earlier of initiation of a Phase 3 clinical study or product approvals in Canada and the U.K., up to $85.0 million in additional regulatory approval milestones for the achievement of U.S. and EU product approvals, and up to $75.0 million in commercial milestone payments based on annual worldwide net product sales. The Company will also pay tiered mid- to high single-digit percentage royalties based on licensed product annual net sales. If the Company receives and resells an FDA priority review voucher, or PRV, in connection with a new drug application approval, GeneTx is entitled to receive a portion of proceeds from the sale or a cash payment from the Company if the Company choses to retain the PRV. As part of the Company's acquisition of GeneTx, the Company assumed a License Agreement with Texas A&M University, or TAMU. Under this agreement, TAMU is eligible to receive from the Company up to $23.5 million upon the achievement of various future milestones, a nominal annual license fee that may increase up to a maximum of $2.0 million, as well as royalties in the mid-single-digits of net sales.

The transaction was accounted as an asset acquisition, as substantially all of the fair value of the gross assets acquired was concentrated in a single identifiable in-process research and development intangible asset. Prior to the achievement of certain development and regulatory milestones, the acquired in-process research and development intangible asset has not yet reached technological feasibility and has no alternative future use. Accordingly, to date, amounts paid to GeneTx, net of cash and working capital acquired, were classified as in-process research and development expense.

Accumulated Other Comprehensive Income

9 Months Ended

Sep. 30, 2024

Other Comprehensive Income (Loss), Net of Tax [Abstract]

Accumulated Other Comprehensive Income

14. Accumulated Other Comprehensive Income

Total accumulated other comprehensive income consisted of the following (in thousands):


	September 30,			December 31,
	2024			2023
Foreign currency translation adjustments	$	(2,177	)	$	(606	)
Unrealized gain on available-for-sale securities		3,073			1,253
Total accumulated other comprehensive income	$	896		$	647

Stock-Based Awards - Additional Information (Details)	Sep. 30, 2024 shares
Amended & Restated 2014 Employee Stock Purchase Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Share reserved for future issuance	6,567,545
Employment Inducement Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Share reserved for future issuance	256,792
2023 Plan
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Share reserved for future issuance	5,916,137

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 35
-Paragraph 1D
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480483/718-10-35-1D

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 35
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CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Operating activities:
Net Income (Loss)	$ (435,798)	$ (483,449)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation	117,866	101,483
Amortization of discount on marketable debt securities, net	(9,818)	(8,894)
Depreciation and amortization	26,464	17,788
Change in fair value of equity investments	(433)	1,492
Non-cash royalty revenue	(67,406)	(42,695)
Non-cash interest expense on liabilities for sales of future royalties	47,519	48,676
Other	(4,246)	3,102
Changes in operating assets and liabilities:
Accounts receivable	(12,958)	(23,790)
Inventory	(8,751)	(4,974)
Other assets	12,269	16,584
Accounts payable, accrued, and other liabilities	607	(16,225)
Net cash used in operating activities	(334,685)	(390,902)
Investing activities:
Purchase of property, plant, and equipment	(6,837)	(42,667)
Purchase of marketable debt securities	(392,680)	(304,789)
Proceeds from sale of marketable debt securities	3,148	47,520
Proceeds from maturities of marketable debt securities	290,675	582,230
Payments for intangible asset	(12,500)	(2,500)
Other	(2,309)	(4,777)
Net cash (used in) provided by investing activities	(120,503)	275,017
Financing activities:
Proceeds from the issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net	380,983	0
Proceeds from the issuance of common stock in connection with at-the-market offering, net	0	53,299
Proceeds from the issuance of common stock under equity plan awards, net of tax	8,072	4,261
Proceeds from issuance of equity from noncontrolling interest	7,000	0
Other	0	(28)
Net cash provided by financing activities	396,055	57,532
Effect of exchange rate changes on cash	(52)	(641)
Net decrease in cash, cash equivalents and restricted cash	(59,185)	(58,994)
Cash, cash equivalents and restricted cash at beginning of period	219,399	137,601
Cash, cash equivalents and restricted cash at end of period	160,214	78,607
Supplemental disclosures of non-cash information:
Acquired lease liabilities arising from obtaining right-of-use assets and property, plant, and equipment	129	23,360
Stock-based compensation capitalized into ending inventory	3,202	2,562
Non-cash interest expense on liabilities for sales of future royalties capitalized during the year into ending property, plant and equipment	$ 0	$ 9,430

GeneTx Acquisition - Additional Information (Details) $ in Millions	1 Months Ended
	Jul. 31, 2022 USD ($)
GeneTx [Member]
Business Acquisition [Line Items]
Business acquisition, consideration transferred	$ 75.0
Outstanding Cash and Cash Equivalents Acquired	15.6
Amount on adjustments for working capital and transaction expenses	0.6
Payments to Acquire Businesses, Gross	91.2
GeneTx [Member] \| Maximum
Business Acquisition [Line Items]
Future contingent milestone payments	190.0
Additional Milestone Payments	30.0
Regulatory Approval Milestones	85.0
Commercial Milestone Payment	75.0
Texas AM University Member \| Maximum
Business Acquisition [Line Items]
Future contingent milestone payments	23.5
Annual license fee payment	$ 2.0

Stock-Based Awards - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock-based compensation expense	$ 41,569	$ 34,877	$ 117,866	$ 101,469
Cost of sales
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock-based compensation expense	428	191	1,113	862
Research and development
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock-based compensation expense	22,569	19,113	64,784	56,371
Selling, general and administrative
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Stock-based compensation expense	$ 18,572	$ 15,573	$ 51,969	$ 44,236

Revenue - Summary of Disaggregation of Total Revenues (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Disaggregation Of Revenue [Line Items]
Total revenues	$ 139,494	$ 98,052	$ 395,353	$ 306,857
North America
Disaggregation Of Revenue [Line Items]
Total revenues	76,914	67,811	223,485	214,232
Latin America
Disaggregation Of Revenue [Line Items]
Total revenues	38,048	17,229	112,155	59,096
Europe, Middle East, and Africa
Disaggregation Of Revenue [Line Items]
Total revenues	22,222	13,012	54,352	32,351
Asia-Pacific
Disaggregation Of Revenue [Line Items]
Total revenues	$ 2,310	$ 0	$ 5,361	$ 1,178

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org/1943274/2147479806/606-10-50-4

Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share

Organization - Additional Information (Details)	9 Months Ended
Organization - Additional Information (Details)	Sep. 30, 2024 Segment
Organization And Nature Of Business [Line Items]
Number of reportable segments	1

Net Loss Per Share (Tables)

9 Months Ended

Sep. 30, 2024

Earnings Per Share [Abstract]


	Three Months Ended September 30,				Nine Months Ended September 30,
	2024		2023		2024		2023
Options to purchase common stock, restricted stock units, and performance stock units		16,667,134		14,585,014		16,235,303		14,117,741
Employee stock purchase plan		65,560		66,735		36,369		37,156
		16,732,694		14,651,749		16,271,672		14,154,897

CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) - USD ($)
$ in Thousands

Sep. 30, 2024

Dec. 31, 2023

Current assets:

Marketable debt securities

^[1]

$ 150,619

$ 213,584

470,989

363,625

Accounts receivable, net

93,301

73,390

Inventory

43,334

33,969

Other assets

41,365

47,616

Total current assets

799,608

732,184

Property, plant, and equipment, net

272,448

290,566

Marketable debt securities

203,086

199,901

Intangible assets, net

164,942

166,271

Goodwill

44,406

Other assets

53,873

57,685

Total assets

1,538,363

1,491,013

Current liabilities:

Accounts payable

35,990

42,114

Accrued liabilities

185,028

196,486

Lease liabilities

10,424

12,595

Liabilities for sales of future royalties

48,731

29,242

Other Liabilities

4,835

Total current liabilities

285,008

280,437

Lease liabilities

23,657

30,574

Deferred tax liabilities

30,058

Liabilities for sales of future royalties

829,686

862,325

Other liabilities

16,127

12,205

Total liabilities

1,184,536

1,215,599

Noncontrolling interest

7,000

Stockholders’ equity:

Preferred stock, par value of $0.001 per share -25,000,000 shares authorized; nil outstanding in 2024 and in 2023

Common stock, par value of $0.001 per share -250,000,000 shares authorized; outstanding - 92,304,317 in 2024 and 82,315,590 in 2023

Treasury stock, at cost, 68,923 in 2024 and 9,559 in 2023

(3,555)

(432)

Deferred compensation obligation

3,555

432

Additional paid-in capital

4,169,298

3,662,346

Accumulated other comprehensive income (loss)

896

647

Accumulated deficit

(3,823,459)

(3,387,661)

Total stockholders’ equity

346,827

275,414

Total liabilities, noncontrolling interest and stockholders' equity

$ 1,538,363

$ 1,491,013

[1]	The Company's Condensed Consolidated Balance Sheet as of September 30, 2024 includes $14.1 million in cash and cash equivalents that can be used only to settle obligations of the consolidated variable interest entity. See Note 6.

Balance Sheet Components - Summary of Accrued Liabilities (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Payables and Accruals [Abstract]
Research, clinical study, and manufacturing expenses	$ 39,752	$ 65,326
Payroll and related expenses	82,924	82,936
Revenue related reserves	32,928	17,029
Other	29,424	31,195
Total accrued liabilities	$ 185,028	$ 196,486

Summary of Significant Accounting Policies - Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash (Details) - USD ($)
$ in Thousands

Sep. 30, 2024

Dec. 31, 2023

Sep. 30, 2023

Dec. 31, 2022

Restricted cash included in other current assets

$ 150,619

^[1]

$ 213,584

^[1]

$ 72,575

$ 6,176

$ 1,976

Restricted Cash and Cash Equivalents, Current, Asset, Statement of Financial Position [Extensible List]

Other assets

Restricted cash included in other non-current assets

$ 3,419

$ 4,056

Restricted Cash and Cash Equivalents, Noncurrent, Asset, Statement of Financial Position [Extensible List]

Other assets

Total cash, cash equivalents, and restricted cash shown in the statements of cash flows

$ 160,214

$ 219,399

$ 78,607

$ 137,601

[1]	The Company's Condensed Consolidated Balance Sheet as of September 30, 2024 includes $14.1 million in cash and cash equivalents that can be used only to settle obligations of the consolidated variable interest entity. See Note 6.

Revenue - Additional Information (Details)	Sep. 30, 2024	Dec. 31, 2023
Customer One
Disaggregation of Revenue [Line Items]
Percentage of gross accounts receivable balance	60.00%	53.00%

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

Investments, Debt and Equity Securities [Abstract]

Balance Sheet Components

9 Months Ended

Sep. 30, 2024

Balance Sheet Components

4. Balance Sheet Components

Cash Equivalents and Marketable Debt Securities

The fair values of cash equivalents and marketable debt securities classified as available-for-sale securities consisted of the following (in thousands):


	September 30, 2024
				Gross Unrealized
		Amortized Cost		Gains		Losses			Estimated Fair Value
Money market funds		$	85,707	$	—	$	—		$	85,707
Certificates of deposit and time deposits			14,142		—		—			14,142
Corporate bonds			422,228		2,177		(31	)		424,374
Commercial paper			22,072		—		—			22,072
Asset-backed securities			243		—		—			243
U.S. Government Treasury and agency securities			228,008		927		—			228,935
Total		$	772,400	$	3,104	$	(31	)	$	775,473


	December 31, 2023
				Gross Unrealized
		Amortized Cost		Gains		Losses			Estimated Fair Value
Money market funds		$	162,289	$	—	$	—		$	162,289
Certificates of deposit and time deposits			17,986		—		—			17,986
Corporate bonds			214,792		711		(337	)		215,166
Commercial paper			20,620		—		—			20,620
Asset-backed securities			2,715		—		(3	)		2,712
U.S. Government Treasury and agency securities			316,160		982		(100	)		317,042
Total		$	734,562	$	1,693	$	(440	)	$	735,815

At September 30, 2024, the remaining contractual maturities of available-for-sale securities were less than three years. There have been no significant realized gains or losses on available-for-sale securities for the periods presented.

Inventory

Inventory consists of the following (in thousands):


	September 30,		December 31,
	2024		2023
Work-in-process	$	20,281	$	18,859
Finished goods		23,053		15,110
Total inventory	$	43,334	$	33,969

Accrued Liabilities

Accrued liabilities consist of the following (in thousands):


	September 30,		December 31,
	2024		2023
Research, clinical study, and manufacturing expenses	$	39,752	$	65,326
Payroll and related expenses		82,924		82,936
Revenue related reserves		32,928		17,029
Other		29,424		31,195
Total accrued liabilities	$	185,028	$	196,486

License and Research Agreements - Schedule of Cost Sharing Payments (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Cost Sharing Payments [Line Items]
Cost of sales	$ 21,021	$ 10,987	$ 59,834	$ 33,158
Kyowa Kirin Collaboration
Cost Sharing Payments [Line Items]
Research and development	(841)	(940)	(2,779)	(4,665)
Selling, general and administrative	(898)	(2,060)	(3,184)	(15,346)
Cost of sales	$ 12,044	$ 4,661	$ 37,952	$ 14,189

Net Loss Per Share - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Common stock equivalents excluded from computation of diluted net loss per share	16,732,694	14,651,749	16,271,672	14,154,897
Options to Purchase Common Stock, RSUs, and PSUs
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Common stock equivalents excluded from computation of diluted net loss per share	16,667,134	14,585,014	16,235,303	14,117,741
Employee Stock Purchase Plan
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Common stock equivalents excluded from computation of diluted net loss per share	65,560	66,735	36,369	37,156

Financial Instruments (Tables)

9 Months Ended

Sep. 30, 2024

Fair Value Disclosures [Abstract]

Summary of Financial Assets Measured on Recurring Basis

The following tables set forth the fair value of the Company’s financial assets and liabilities remeasured on a recurring basis based on the three-tier fair value hierarchy (in thousands):


	September 30, 2024
	Level 1		Level 2		Level 3		Total
Financial Assets:
Money market funds	$	85,707	$	—	$	—	$	85,707
Certificates of deposit and time deposits		—		14,142		—		14,142
Corporate bonds		—		424,374		—		424,374
Commercial paper		—		22,072		—		22,072
Asset-backed securities		—		243		—		243
U.S. Government Treasury and agency securities		30,781		198,154		—		228,935
Investment in Solid common stock		3,637		—		—		3,637
Deferred compensation assets		—		14,609		—		14,609
Total financial assets	$	120,125	$	673,594	$	—	$	793,719

Financial Liabilities:
Deferred compensation liabilities	$	—	$	14,902	$	—	$	14,902


	December 31, 2023
	Level 1		Level 2		Level 3		Total
Financial Assets:
Money market funds	$	162,289	$	—	$	—	$	162,289
Certificates of deposit and time deposits		—		17,986		—		17,986
Corporate bonds		—		215,166		—		215,166
Commercial paper		—		20,620		—		20,620
Asset-backed securities		—		2,712		—		2,712
U.S. Government Treasury and agency securities		57,437		259,605		—		317,042
Investment in Solid common stock		3,204		—		—		3,204
Deferred compensation assets		—		10,220		—		10,220
Total financial assets	$	222,930	$	526,309	$	—	$	749,239

Financial Liabilities:
Deferred compensation liabilities	$	—	$	10,365	$	—	$	10,365

Financial Instruments - Summary of Financial Assets Measured on Recurring Basis (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	$ 793,719	$ 749,239
Level 1
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	120,125	222,930
Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	673,594	526,309
Money Market Funds
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	85,707	162,289
Money Market Funds \| Level 1
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	85,707	162,289
Asset-backed Securities
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	243	2,712
Asset-backed Securities \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	243	2,712
Certificates of Deposit and Time Deposits
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	14,142	17,986
Certificates of Deposit and Time Deposits \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	14,142	17,986
Corporate Bonds
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	424,374	215,166
Corporate Bonds \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	424,374	215,166
Commercial Paper
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	22,072	20,620
Commercial Paper \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	22,072	20,620
U.S. Government Treasury and Agency Securities
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	228,935	317,042
U.S. Government Treasury and Agency Securities \| Level 1
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	30,781	57,437
U.S. Government Treasury and Agency Securities \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	198,154	259,605
Investments in Solid Common Stock
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	3,637	3,204
Investments in Solid Common Stock \| Level 1
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	3,637	3,204
Deferred Compensation Assets
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	14,609	10,220
Deferred Compensation Assets \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	14,609	10,220
Deferred Compensation Liabilities
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	14,902	10,365
Deferred Compensation Liabilities \| Level 2
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total fair value	$ 14,902	$ 10,365

Balance Sheet Components - Summary of Cash Equivalents and Marketable Debt Securities Classified as Available For Sale Securities (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	$ 772,400	$ 734,562
Gross Unrealized Gains	3,104	1,693
Gross Unrealized Losses	(31)	(440)
Estimated Fair Value	775,473	735,815
Money Market Funds
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	85,707	162,289
Estimated Fair Value	85,707	162,289
Asset-backed Securities
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	243	2,715
Gross Unrealized Losses		(3)
Estimated Fair Value	243	2,712
Certificates of Deposit and Time Deposits
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	14,142	17,986
Estimated Fair Value	14,142	17,986
Corporate Bonds
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	422,228	214,792
Gross Unrealized Gains	2,177	711
Gross Unrealized Losses	(31)	(337)
Estimated Fair Value	424,374	215,166
Commercial Paper
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	22,072	20,620
Estimated Fair Value	22,072	20,620
U.S. Government Treasury and Agency Securities
Schedule Of Available For Sale Securities [Line Items]
Amortized Cost	228,008	316,160
Gross Unrealized Gains	927	982
Gross Unrealized Losses		(100)
Estimated Fair Value	$ 228,935	$ 317,042

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Pay vs Performance Disclosure
Net Income (Loss)	$ (133,516)	$ (159,649)	$ (435,798)	$ (483,449)

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

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Accumulated Other Comprehensive Income - Schedule of Total Accumulated Other Comprehensive Income (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract]
Foreign currency translation adjustments	$ (2,177)	$ (606)
Unrealized gain on available-for-sale securities	3,073	1,253
Total accumulated other comprehensive income	$ 896	$ 647

Amount, after tax, of accumulated increase (decrease) in equity from transaction and other event and circumstance from nonowner source.

Summary of Significant Accounting Policies (Tables)

9 Months Ended

Sep. 30, 2024

Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Condensed Consolidated Statement of Cash Flows (in thousands):


	September 30,
	2024		2023
Cash and cash equivalents	$	150,619	$	72,575
Restricted cash included in other current assets		6,176		1,976
Restricted cash included in other non-current assets		3,419		4,056
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows	$	160,214	$	78,607

Stock-Based Awards

9 Months Ended

Sep. 30, 2024

Share-Based Payment Arrangement [Abstract]

Stock-Based Awards

. Stock-Based Awards

As of September 30, 2024, there were 5,916,137 shares available under the 2023 Incentive Plan, 6,567,545 shares available under the Amended & Restated 2014 Employee Stock Purchase Plan, and 256,792 shares available under the Employment Inducement Plan for the future issuance of equity awards.

The table below sets forth the stock-based compensation expense for the periods presented (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2024		2023		2024		2023
Cost of sales	$	428	$	191	$	1,113	$	862
Research and development		22,569		19,113		64,784		56,371
Selling, general and administrative		18,572		15,573		51,969		44,236
Total stock-based compensation expense	$	41,569	$	34,877	$	117,866	$	101,469

Investment in Amlogenyx. Inc. (Tables)

9 Months Ended

Sep. 30, 2024

Investments, All Other Investments [Abstract]

Changes in the Carrying Value of Noncontrolling Interest

Changes in the carrying value of noncontrolling interest for the nine months ended September 30, 2024 are as follows:


	Noncontrolling Interest
As of December 31, 2023	$	—
Issuance of equity from noncontrolling interest		7,000
As of September 30, 2024	$	7,000

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Statement of Comprehensive Income [Abstract]
Net loss	$ (133,516)	$ (159,649)	$ (435,798)	$ (483,449)
Other comprehensive income (loss):
Foreign currency translation adjustments	(1,481)	(249)	(1,571)	188
Unrealized gain on available-for-sale debt securities	3,472	1,103	1,820	4,141
Other comprehensive income:	1,991	854	249	4,329
Total comprehensive loss	$ (131,525)	$ (158,795)	$ (435,549)	$ (479,120)

Amount after tax of increase (decrease) in equity from transactions and other events and circumstances from net income and other comprehensive income, attributable to parent entity. Excludes changes in equity resulting from investments by owners and distributions to owners.

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

Amount after tax, before reclassification adjustments of gain (loss) on foreign currency translation adjustments, foreign currency transactions designated and effective as economic hedges of a net investment in a foreign entity and intra-entity foreign currency transactions that are of a long-term-investment nature.

Amount after tax and reclassification adjustments of other comprehensive income (loss).

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Organization, Consolidation and Presentation of Financial Statements [Abstract]

Organization

9 Months Ended

Sep. 30, 2024

Organization

1. Organization

Ultragenyx Pharmaceutical Inc., or the Company, is a biopharmaceutical company incorporated in Delaware.

The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultrarare genetic diseases. The Company operates as one reportable segment and has four commercially approved products.

Crysvita® (burosumab) is approved in the United States, or U.S., the European Union, or EU, and certain other regions for the treatment of X-linked hypophosphatemia, or XLH, in adult and pediatric patients one year of age and older. Crysvita is also approved in the U.S. and certain other regions for the treatment of fibroblast growth factor 23, or FGF23-related hypophosphatemia in tumor-induced osteomalacia, or TIO, associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.

Mepsevii® (vestronidase alfa) is approved in the U.S., the EU and certain other regions, as the first medicine for the treatment of children and adults with mucopolysaccharidosis VII, or MPS VII, also known as Sly syndrome.

Dojolvi® (triheptanoin) is approved in the U.S. and certain other regions for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders, or LC-FAOD.

Evkeeza® (evinacumab) is approved in the U.S. and the European Economic Area, or EEA, and Japan for the treatment of homozygous familial hypercholesterolemia, or HoFH. The Company has exclusive rights to commercialize Evkeeza® (evinacumab) outside of the U.S.

In addition to the approved products, the Company has the following ongoing clinical development programs:

•

UX111 (formerly ABO-102) is an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease;

•

DTX401 is an adeno-associated virus 8, or AAV8, gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia, or GSDIa;

•

DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase, or OTC deficiency, the most common urea cycle disorder;

•

UX143 (setrusumab), which is subject to the Company’s collaboration agreement with Mereo BioPharma 3, or Mereo, is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway and inhibits the activity of bone-forming cells for the treatment of patients with Osteogenesis Imperfecta, or OI;

•

GTX-102 is an antisense oligonucleotide, or ASO for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene; and

•

UX701 is an adeno-associated virus 9, or AAV9, gene therapy designed to deliver stable expression of a truncated version of the ATP7B copper transporter following a single intravenous infusion to improve copper distribution and excretion from the body and reverse pathological findings of Wilson liver disease.

The Company has sustained operating losses and expects such annual losses to continue over the next several years. The Company’s ultimate success depends on the outcome of its research and development and commercialization activities. Through September 30, 2024, the Company has relied primarily on its sale of equity securities, its revenues from commercial products, its sale of future royalties, and strategic collaboration arrangements to finance its operations. The Company may need to raise additional capital to fully implement its business plans through the issuance of equity, borrowings, or strategic alliances with partner companies. However, if such financing is not available at adequate levels, the Company would need to reevaluate its operating plans.

License and Research Agreements

9 Months Ended

Sep. 30, 2024

Research Grant Agreement [Abstract]

License and Research Agreements

. License and Research Agreements

Kyowa Kirin Co., Ltd.

In August 2013, the Company entered into a collaboration and license agreement with Kyowa Kirin Co., Ltd., or KKC. Under the terms of this collaboration and license agreement, as amended, the Company and KKC collaborate on the development and commercialization of Crysvita in the field of orphan diseases in the U.S. and Canada, or the Profit-Share Territory, and in the European Union, United Kingdom, and Switzerland, or the European Territory, and the Company has the right to develop and commercialize such products in the field of orphan diseases in Mexico and Central and South America, or Latin America.

Development Activities

In the field of orphan diseases, except for ongoing studies being conducted by KKC, the Company was the lead party for development activities in the Profit-Share Territory and in the European Territory until the applicable transition date. The Company shared the costs for development activities in the Profit-Share Territory and the European Territory conducted pursuant to the development plan before the applicable transition date equally with KKC. In April 2023, which was the transition date for the Profit-Share Territory, KKC became the lead party and became responsible for the costs of the subsequent development activities. However, the Company will continue to equally share in the costs of the studies with KKC that commenced prior to the applicable transition date.

The collaboration and license agreements are within the scope of ASC 808, which provides guidance on the presentation and disclosure of collaborative arrangements.

Collaboration and Royalty Revenue for Sales in the Profit-Share Territory

The Company and KKC shared commercial responsibilities and profits in the Profit-Share Territory until April 2023. Under the collaboration agreement, KKC manufactured and supplied Crysvita for commercial use in the Profit-Share Territory and charged the Company a transfer price of 30% of net sales in 2023, and 35% prior to December 31, 2022. The remaining profit or loss after supply costs from commercializing products in the Profit-Share Territory was shared between the Company and KKC on a 50/50 basis until April 2023. In April 2023, commercialization responsibilities for Crysvita in the Profit-Share Territory transitioned to KKC. Thereafter, the Company is entitled to receive a tiered double-digit revenue share from the mid-20% range up to a maximum rate of 30%.

In 2022, the Company entered into an amendment to the collaboration agreement which granted the Company the right to continue to support KKC in commercial field activities in the U.S. through April 2024, subject to the limitations and conditions set forth in the amendment. The parties subsequently mutually agreed to extend the Company's right to continue to support KKC in commercial field activities in the U.S. through December 31, 2024, and as a result, the Company will continue to support commercial field efforts in the U.S. through a cost share arrangement through December 2024. After December 31, 2024, the Company’s rights to promote Crysvita in the U.S. will be limited to medical geneticists and the Company will solely bear its expenses for the promotion of Crysvita in the Profit-Share Territory.

As KKC was the principal in the sale transaction with the customer during the profit-share period, the Company recognized a pro-rata share of collaboration revenue, net of transfer pricing, in the period the sale occurred. The Company concluded that its portion of KKC’s sales in the Profit-Share Territory prior to April 2023 was analogous to a royalty and therefore recorded its share as collaboration revenue, similar to a royalty. Starting in April 2023, the Company began to record the royalty revenue as the underlying sales occurred.

In July 2022, the Company sold to OMERS its right to receive 30% of the future royalty payments due to the Company based on net sales of Crysvita in the U.S. and Canada, subject to a cap, beginning in April 2023, as further described in Note 9. The Company records this revenue as royalty revenue.

Product Sales Revenue for Other Territories

The Company is responsible for commercializing Crysvita in Latin America and Turkey. The Company is considered the principal in these territories as the Company controls the product before it is transferred to the customer. Accordingly, the Company records revenue on a gross basis for the sale of Crysvita once the product is delivered and the risk and title of the product is transferred to the distributor. KKC has the option to assume responsibility for commercialization efforts in Turkey from the Company, after a certain minimum period.

Under the collaboration agreement, KKC manufactures and supplies Crysvita, which is purchased by the Company for sales in Latin American territories and charges the Company a transfer price of 30% of net sales. The transfer price on these sales was 35% prior to December 31, 2022. The Company also pays to KKC a low single-digit royalty on net sales in Latin America.

Royalty Revenue for Sales in the European Territory

KKC has the commercial responsibility for Crysvita in the European Territory. In December 2019, the Company sold its right to receive royalty payments based on sales in the European Territory to Royalty Pharma, effective January 1, 2020, as further described in Note 9. Prior to the Company’s sale of the royalty, the Company received a royalty of up to 10% on net sales in the European Territory, which was recognized as the underlying sales occur. Beginning in 2020, the Company records the royalty revenue as non-cash royalty revenues.

Total Crysvita revenue was as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2024		2023		2024		2023
Revenue in Profit-Share Territory:
Royalty revenue	$	39,189	$	35,160	$	114,402	$	64,221
Non-cash royalty revenue		16,796		15,070		49,030		27,524
Collaboration revenue		—		—		—		69,705
Total revenue in Profit-Share Territory		55,985		50,230		163,432		161,450
Product sales		35,604		19,200		112,294		57,318
Non-cash royalty revenue in European Territories		6,258		5,473		18,376		15,171
Total Crysvita revenue	$	97,847	$	74,903	$	294,102	$	233,939

Collaboration Cost Sharing and Payments

Under the collaboration agreement, KKC and the Company share certain development and commercialization costs and as a result, the Company was reimbursed for these costs and operating expenses were reduced. Additionally, KKC is owed a transfer price fee and royalties on certain revenues and the Company recorded amounts owed to KKC in cost of sales. These amounts were recognized in the Company’s Condensed Consolidated Statements of Operations in connection with the collaboration agreement with KKC as follows (in thousands):


	Three Months Ended September 30,						Nine Months Ended September 30,
	2024			2023			2024			2023
Research and development	$	(841	)	$	(940	)	$	(2,779	)	$	(4,665	)
Selling, general and administrative		(898	)		(2,060	)		(3,184	)		(15,346	)
Cost of sales		12,044			4,661			37,952			14,189

Collaboration Receivable and Payable

The Company had accounts receivable from KKC in the amount of $55.9 million and $39.2 million from profit-share revenue and royalties, other receivables of $1.8 million and $1.1 million recorded in other current assets, and accrued liabilities of $11.7 million and $5.3 million from inventory, commercial, and development activity reimbursements, as of September 30, 2024 and December 31, 2023, respectively.

Mereo

In December 2020, the Company entered into a License and Collaboration Agreement with Mereo to collaborate on the development of setrusumab. Under the terms of the agreement, the Company leads future global development of setrusumab in both pediatric and adult patients with Osteogenesis Imperfecta, or OI. The Company was granted an exclusive license to develop and commercialize setrusumab in the U.S., Turkey, and the rest of the world, excluding the European Economic Area, United Kingdom, and Switzerland, or the Mereo Territory, where Mereo retains commercial rights. Each party is responsible for post-marketing commitments and commercial supply in their respective territories.

Upon the closing of the transactions under the License and Collaboration Agreement with Mereo in January 2021, the Company made a payment of $50.0 million to Mereo. In July 2023, the Company made a $9.0 million payment to Mereo upon the achievement of a clinical milestone. Going forward, the Company may be required to make payments of up to $245.0 million upon the achievement of certain clinical, regulatory, and commercial milestones. The Company will pay for all global development costs as well as tiered double-digit percentage royalties to Mereo on net sales in the U.S., Turkey, and the rest of the world, and Mereo will pay the Company a fixed double-digit percentage royalty on net sales in the Mereo Territory.

Although Mereo is a VIE, the Company is not the primary beneficiary as it does not have the power to direct the activities that would most significantly impact the economic performance of Mereo. Prior to the achievement of certain development milestones, all consideration paid to Mereo represents rights to potential future benefits associated with Mereo’s in-process research and development activities, which have not reached technological feasibility and have no alternative future use.

Regeneron

In January 2022, the Company announced a collaboration with Regeneron Pharmaceuticals, or Regeneron, to commercialize Evkeeza for HoFH outside of the U.S. Evkeeza is approved in the U.S., where it is marketed by Regeneron, and in the EEA and Japan as a first-in-class therapy for use together with diet and other low-density lipoprotein-cholesterol-lowering therapies to treat adults and adolescents aged 12 years and older with HoFH. Pursuant to the terms of the agreement, the Company received the rights to develop, commercialize and distribute the product for HoFH in countries outside of the U.S. The Company is obligated to pay up to $63.0 million in future milestone payments, contingent upon the achievement of certain regulatory and sales milestones. The Company may share in certain costs for global trials led by Regeneron and also received the right to opt into other potential indications.

The collaboration agreement is within the scope of ASC 808 which provides guidance on the presentation and disclosure of collaborative arrangements. As the Company would be the principal in future sale transactions with the customer, the Company recognizes product sales and cost of sales in the period the related sales occur and the related revenue recognition criteria are met. Under the collaboration agreement, Regeneron supplies the product and charges the Company a transfer price from the low 20% range up to 40% on net sales, which is recognized as cost of sales in the Company’s Condensed Consolidated Statement of Operations.

The Company paid Regeneron a $30.0 million upfront payment upon the closing of the transaction in January 2022, a $10.0 million regulatory milestone payment in January 2024, and a $2.5 million regulatory milestone payment in May 2024. As these payments are for the Company’s use of intellectual property for Evkeeza for HoFH, they were recorded as intangible assets.

Under the collaboration agreement, the Company recognized $0.3 million, net, in development expense for the three months ended September 30, 2024, and recognized an offset of $2.5 million, net, to development expense for the nine months ended September 30, 2024, respectively, recorded as an increase and offset, respectively, to research and development expense. The Company recognized $1.9 million, net, and $6.9 million, net, in development expense under this agreement for the three and nine months ended September 30, 2023, respectively, recorded as research and development expense. The Company had collaboration payables for this arrangement included in accrued liabilities of $2.5 million and $10.6 million as of September 30, 2024 and December 31, 2023, respectively. Additionally, Regeneron is owed a transfer price fee and royalties of $2.7 million and $5.4 million for the three and nine months ended September 30, 2024, respectively, and $0.2 million and $0.4 million for the three and nine months ended September 30, 2023, respectively, recorded in cost of sales in the financial statements.

Other Arrangements

The Company has also entered into several collaborations and/or licensing arrangements in prior periods. Except as disclosed above, there have been no material changes in these arrangements during the three and nine months ended September 30, 2024 as compared to those disclosed in "Note 8. License and Research Agreements" to the Consolidated Financial Statements in the Annual Report.

Under the financial terms of these arrangements, the Company may be required to make payments upon achievement of developmental, regulatory, and commercial milestones, which could be significant. Future milestone payments, if any, will be

reflected in the financial statements upon the occurrence of the contingent event. In addition, the Company may be required to pay royalties on future sales if products related to these arrangements are commercialized. The payment of these amounts, however, is contingent upon the occurrence of various future events, which have a high degree of uncertainty.

As described in the Annual Report, the Company holds an equity interest in Solid in connection with its collaboration arrangement. The changes in the fair value of the Company’s equity investment in the common stock of Solid were as follows (in thousands):


	Common stock
As of December 31, 2022	$	2,807
Change in fair value		397
As of December 31, 2023		3,204
Change in fair value		433
As of September 30, 2024	$	3,637

Summary of Significant Accounting Policies

9 Months Ended

Sep. 30, 2024