FDA approves Ultragenyx’s drug for rare form of rickets


The U.S. Food and Drug Administration has approved Ultragenyx Pharmaceutical Inc’s treatment for a rare and inherited form of rickets, a condition that causes weak bones, the agency said on Tuesday.

California-based Ultragenyx’s shares rose 4.6 percent to $56.31 in afternoon trading.

The drug, Crysvita, was approved to treat x-linked hypophosphatemia in children and adults.

The condition causes bowed legs, short stature and bone pain in children, and affects about 3,000 children and 12,000 adults in the United States.

Some adults with the disease experience joint pain, impaired mobility, tooth abscesses and hearing loss.

The FDA also gave Ultragenyx a “rare pediatric disease voucher,” which can be redeemed at a later date to receive a priority review from the agency for another product.


Reporting by Manas Mishra in Bengaluru; Editing by Arun Koyyur


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