Jan. 13, 2020: Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd. announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) on December 18, 2019, 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 companies expect to hear back from FDA on submission acceptance and review designation in February 2020.

“Around half of patients suffering  with TIO have tumors that cannot be surgically removed, leaving them with no other current treatment options,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx.

“We look forward to continuing to work closely with the FDA during the review process, with the goal of bringing Crysvita to patients with TIO in the U.S.”

The sBLA package includes data from the two single-arm Phase 2 studies, a 144-week study in 14 adult patients conducted by the Ultragenyx in the U.S. and an 88-week study in 13 adult patients conducted by the Kyowa Kirin in Japan and South Korea.

In both these studies, Crysvita was associated with increases in serum phosphorus and serum 1,25-dihydroxyvitamin D levels. Increased phosphate levels will led to improvements in osteomalacia, mobility and vitality also bone scans demonstrated in an increase in healed fractures and a decrease in new fractures during Crysvita treatment.

FDA also approved Crysvita for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients six months of age and older, and by Health Canada and Brazil’s National Health Surveillance Agency (ANVISA) for the treatment of XLH in the adult and pediatric patients one year of age and older.

Crysvita is approved by Japan’s Ministry of Health, Labor and Welfare (MHLW) for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia.

The medicine has received the 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 the growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency. https://fda.einnews.com/pr_news/507058520/ultragenyx-and-kyowa-kirin-announce-submission-of-supplemental-biologics-license-application-to-u-s-fda-for-crysvita-burosumab-for-tumor-induced