Ultragenyx and Kyowa Kirin Announce Publication of Phase 2 Study Results Demonstrating that Crysvita® (burosumab) Improved Outcomes in Children with X-linked Hypophosphatemia in the New England Journal of Medicine
XLH is a rare, chronic progressive musculoskeletal disorder that affects children and adults. Crysvita is the first treatment to target the underlying pathophysiology of XLH – excess production of fibroblast growth factor 23 (FGF23), a hormone that regulates phosphate excretion and active vitamin D production by the kidney. In the NEJM article entitled “Burosumab Therapy in Children with X-Linked Hypophosphatemia” the authors state, “Inhibition of FGF-23 activity with burosumab, a recombinant human lgG1 monoclonal antibody, was associated with an increase in renal tubular phosphate reabsorption and the correction of hypophosphatemia in children with X-linked hypophosphatemia. The improvement in phosphate metabolism corresponded to a decrease in the severity of rickets. The healing of rickets probably contributed to concurrent improvements in growth and physical activity and a reduction in pain.”
“This study was the natural extension of recent discoveries related to disease mechanisms in XLH, and in particular FGF23’s role as a mediator of renal phosphate metabolism,” said
“XLH causes a significant burden on children and their families and the consequences of the disease can affect patients for their entire lives,” said
“I am delighted that these results have been accepted by the
Phase 2 Study Design
The randomized, multicenter, open-label, dose-finding Phase 2 study enrolled 52 children aged 5 to 12 years with XLH at nine sites in
About X-Linked Hypophosphatemia (XLH)
XLH is a rare, hereditary, progressive and lifelong skeletal disorder characterized by renal phosphate wasting caused by excess FGF23 production. It affects both children and adults. In children, XLH causes rickets that leads to lower-extremity deformity, delayed growth and decreased height. Adults with XLH have an increased risk of fractures.
Crysvita is a recombinant fully human monoclonal IgG1 antibody, discovered by
INDICATION (IN THE U.S.)
Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older.
IMPORTANT SAFETY INFORMATION
Crysvita should not be taken if:
- An oral phosphate supplement and a specific form of vitamin D supplement are taken
- Phosphorus levels from a blood sample are within or above the normal range for age
- Kidney problems are present
What is the most important information to know about Crysvita?
- Some patients developed allergic reactions (rash and hives) while taking Crysvita. Doctors will monitor for symptoms of an allergic reaction while Crysvita is taken.
- High levels of phosphorus in the blood have been reported in some patients taking Crysvita. This may be related to a risk of high calcium levels in the kidneys. Doctors will collect samples to monitor levels.
Administration of Crysvitamay result in reactions at the injection site, such as hives, reddening of the skin, rash, swelling, bruising, pain, severe itching of the skin, and collection of blood outside of a blood vessel (hematoma).
What are the possible side effects of Crysvita?
|•||The most common adverse reactions that were seen in children with XLH are:|
|•||The most common adverse reactions that were seen in adults with XLH are:|
|•||Narrowing of the spaces within the spine is common in adults with XLH and pressure on the spinal cord has been reported in adults taking Crysvita. It is not known if taking Crysvita worsens the narrowing of the spaces within the spine or the pressure on the spinal cord.
Before taking Crysvita, doctors should be informed about all medical conditions, including if:
- One is pregnant, thinks she may be pregnant, or plans to become pregnant. There is not enough experience to know if Crysvita may harm an unborn baby. Report pregnancies to the Ultragenyx Adverse Event reporting line at 1-888-756-8657.
- One is breastfeeding or plans to breastfeed. There is not enough experience to know if Crysvita passes into breast milk. Women should talk with their doctors about the best way to feed their babies while taking Crysvita.
While taking Crysvita, doctors should be informed if one experiences:
- An allergic reaction such as rash or hives
- A rash, swelling, bruising or other reaction at the injection site
- New or worsening restless leg syndrome
These are not all the possible side effects of Crysvita. Doctors should be contacted for medical advice about side effects.
Side effects may be reported to the
Please see full Prescribing Information for additional Important Safety Information.
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.
About Kyowa Kirin
Kyowa Kirin International PLC is a wholly owned subsidiary of
You can learn more about the business at: www.kyowa-kirin.com.
Ultragenyx Forward-Looking Statements
Except for the historical information contained herein, the matters set forth in this press release, including statements relating to the expected importance of a therapeutic approach as an advance in the therapy of XLH, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the
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Source: Ultragenyx Pharmaceutical Inc.