FDA Approves First Therapy for Rare Disease that Causes Low Phosphate Blood Levels, Bone Softening

發(fā)布時間： 2020-06-29 閱讀：782次

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June 18, 2020-- Today, the U.S. Food and Drug Administration approved Crysvita (burosumab-twza) injection to treat patients age two and older with tumor-induced osteomalacia (TIO), a rare disease that is characterized by the development of tumors that cause weakened and softened bones. The tumors associated with TIO release a peptide hormone-like substance known as fibroblast growth factor 23 (FGF23) that lowers phosphate levels.

FGF23 regulates levels of phosphate, an electrolyte that plays important roles in bone maintenance, energy production by cells and nerve function. When there is not enough phosphate in the body, bones begin to soften and weaken, causing osteomalacia (marked softening of bones).

“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.”