FDA Approves Burosumab-Twza For Rare Form Of Rickets.

reports the Food and Drug Administration approved Ultragenyx Pharmaceutical Inc.’s Crysvita (burosumab-twza) “to treat x-linked hypophosphatemia in children and adults.” The FDA gave Ultragenyx a “rare pediatric disease voucher,” because the disease is “a rare and inherited form of rickets.” Medscape (4/17, Brooks, Subscription Publication) reports Dr. Julie Beitz, the director of the FDA’s Center for Drug Evaluation and Research’s Office of Drug Evaluation III, said, “XLH differs from other forms of rickets in that vitamin D therapy is not effective. This is the first FDA-approved medication for the treatment of XLH and a real breakthrough for those living with this serious disease.” https://www.reuters.com/article/us-ultragenyx-fda/fda-approves-ultragenyxs-drug-for-rare-form-of-rickets-idUSKBN1HO2NW