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Ultragenyx and Kyowa Kirin Announce U.S. FDA Approval of Crysvita® (burosumab) for the Treatment of Tumor-Induced Osteomalacia (TIO)
First Approved Therapy in the United States for Patients with TIO Who Cannot Undergo Surgical Removal of Tumors TIO is Second FDA-Approved Indication for
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"First Approved Therapy in the United States for Patients with TIO Who Cannot Undergo Surgical Removal of Tumors\n TIO is Second FDA-Approved Indication for Crysvita, Which is Also Approved for the Treatment of X-linked Hypophosphatemia (XLH) NOVATO, Calif. and TOKYO, June 18, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. Food and Drug Administration (FDA) has approved Crysvita® (burosumab) for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older. Crysvita is a human antibody that blocks excess activity of FGF23, a hormone that causes phosphate urinary excretion and suppresses active vitamin D production by the kidney. “For approximately half of all individuals with TIO, surgical removal of the tumors is not possible, leaving these patients with no other treatment options. The FDA approval of Crysvita marks the first treatment option that addresses the cause of the severe hypophosphatemia and osteomalacia resulting from these rare tumors,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We plan to leverage our experience and existing infrastructure with Crysvita in X-linked hypophosphatemia to bring this important medicine to patients living with the rare, painful and debilitating disorder of TIO.” “Since its approval, Crysvita has meant a great deal to patients and families that previously had no other treatment options. We are proud of the work that has been done to advance this discovery from our labs, through a robust clinical research program, and through the FDA’s priority review process, to make this treatment available to patients with TIO,” said Gary Zieziula, President, North America for Kyowa Kirin. “Our commitment to meeting the needs of patients with rare and serious diseases remains steadfast and we will continue to partner with the Ultrage...