Ultragenyx Announces Approval of Crysvita (burosumab) in Brazil for the Treatment of X-linked Hypophosphatemia (XLH) in Adults and Children

Globe Newswire 26-Mar-2019 8:31 AM

NOVATO, Calif., March 26, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Brazil's National Health Surveillance Agency (ANVISA) has approved Crysvita® (burosumab) for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older.

"This approval of Crysvita offers patients in Brazil the first treatment option that targets the underlying cause of XLH, and also marks the first Crysvita approval in Latin America," said Eduardo Thompson, Senior Vice President and Regional Head, Latin America at Ultragenyx. "Crysvita is now approved in three key regions of the world including North America, Europe, and now the first country in Latin America, all in just over a year."

Crysvita is also approved by the U.S. Food and Drug Administration (FDA) and by Health Canada for the treatment of XLH in adult and pediatric patients one year of age and older, and has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.

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.

About Crysvita
Crysvita is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Hakko Kirin, against the phosphaturic hormone fibroblast growth factor 23 (FGF23). FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in XLH is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

Kyowa Hakko Kirin, Kyowa Kirin International, a wholly owned subsidiary of Kyowa Hakko Kirin, and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally, based on the collaboration and license agreement between Kyowa Hakko Kirin and Ultragenyx.

INDICATION (IN THE U.S)

Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 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 Crysvita may 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 seen in children with XLH:

• • Headache
  • Injection site reaction
  • Vomiting
  • Fever
  • Pain in arms and legs
  • Decreased vitamin D levels
  • Rash
  • Toothache
  • Muscle pain
  • Tooth infection
  • Dizziness

• The most common adverse reactions seen in adults with XLH:

• • Back pain
  • Headache
  • Tooth infection
  • Restless leg syndrome
  • Decreased vitamin D levels
  • Dizziness
  • Constipation
  • 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:

• Pregnant, thinks one may be pregnant, or plan to become pregnant. There is not enough experience to know if Crysvita may harm an unborn baby. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-888-756-8657.
• Breastfeeding or plan 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 FDA at (800) FDA-1088 or www.fda.gov/medwatch. Side effects may also be reported to Kyowa Kirin, Inc. at 1-888-756-8657.

Please see full Prescribing Information for additional Important Safety Information.

About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, 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.

Contact Ultragenyx Pharmaceutical Inc.
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Danielle Keatley
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