On June 18, 2020, Ultragenyx Pharmaceutical (NASDAQ: RARE) and Kyowa Kirin Co., Ltd. issued a joint announcement that the U.S. 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.
“Treatment for TIO focuses on identifying and removing the tumor that causes the disease. However, when that is not possible, Crysvita can help increase the levels of phosphate in the blood,” said Theresa E. Kehoe, M.D., acting director of the Division of General Endocrinology in the FDA’s Center for Drug Evaluation and Research. “As the first FDA-approved therapy to treat this debilitating disease, today’s action is an important step in finding treatment options for patients living with TIO whose tumor cannot be found or removed.”
“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 Ultragenyx team to address these needs with urgency.”
TIO is a rare disease caused by typically benign, slow-growing tumors that produce excess levels of FGF23, which is involved in phosphate reabsorption.
Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) shares are trading around $72 on Thursday, just a $4 short of 52-week high.