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Ultragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) in Tumor-Induced Osteomalacia (TIO)
NOVATO, Calif. and TOKYO, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"NOVATO, Calif. and TOKYO, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., (Kyowa Kirin, TYO: 4151) today announced plans to submit a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The decision to submit follows the completion of a pre-sBLA meeting with the FDA and agreement on the filing package. The submission of the Crysvita sBLA is planned for the first half of 2020 and will be based on the current clinical data package.\n\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n\n “Based on productive discussions with the FDA, we will be moving forward expeditiously with an sBLA filing for Crysvita in tumor-induced osteomalacia,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We look forward to working with the agency during the review process, and we are committed to getting this therapy to patients with this serious disease with significant unmet medical need.” The BLA package will include data from two single-arm Phase 2 studies, a 144-week Phase 2 study in 14 adult patients conducted by Ultragenyx in the U.S. and an 88-week Phase 2 study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea. In both studies, Crysvita was associated with increases in serum phosphorus and serum 1,25-dihydroxyvitamin D levels. Increased phosphate levels led to improvements in osteomalacia, mobility, and vitality. Bone scans also demonstrated an increase in healed fractures and decrease in new fractures during Crysvita treatment. Adverse events generally reflected the patients’ underlying disease, and there were no serious treatment-related adverse events during the studies. Crysvita is approved by the U.S. FDA, Health Canada, and Brazil’s National Health Surveillance Agency (ANVISA) for the treatment of X-linked hypophosphatemia (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 radiog...