Ultragenyx and Kyowa Kirin Announce FDA Approval of Crysvita® (burosumab-twza) for the Treatment of Children and Adults with X–Linked Hypophosphatemia (XLH)
First Approved Therapy for XLH in the U.S.; Only Treatment that Targets the Underlying Cause of this Rare, Hereditary, Lifelong Disease
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“Patients now have an approved breakthrough therapy that can help correct the underlying disease, transforming the treatment of XLH and reducing related bone disease in both children and adults living with this disease,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “This milestone represents Ultragenyx’s second approved therapy in less than six months and validates our strategy to rapidly transform good science into effective therapies for rare diseases. This approval would not have been possible without the patients, their families and clinicians who participated and I would like to thank them for their commitment and dedication.”
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 designed to bind the excess FGF23 in these patients, normalizing phosphorus levels, improving bone mineralization, improving rickets in children and healing fractures in adults.
“The approval of Crysvita is truly a watershed moment for patients with X-linked hypophosphatemia as it is the first therapy directed toward correction of renal phosphate wasting,” said
With this approval, the
In order to support patients, Ultragenyx has launched UltraCare™, a support service that will provide ongoing support to patients and caregivers. UltraCare will help patients understand insurance coverage and assist in finding financial support for both medication and administration of medication. Dedicated in-house UltraCare Guides are available Monday through Friday from
Efficacy Results in Clinical Studies
For the pediatric XLH population, the
For the adult XLH indication, the
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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:
• Injection site reaction
• Pain in arms and legs
• Decreased vitamin D levels
• Muscle pain
• Tooth infection
- The most common adverse reactions that were seen in adults with XLH are:
• Back pain
• Tooth infection
• Restless leg syndrome
• Decreased vitamin D levels
• Phosphorus levels increased in the blood
- 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 therapies 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 approved and investigational therapies 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.
Except for the historical information contained herein, the matters set forth in this press release, including statements relating to Ultragenyx's plans or expectations regarding the availability of Crysvita, 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 Annual Report on Form 10-K filed with the
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Source: Ultragenyx Pharmaceutical Inc.