FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A News provided by Share this article Share this article SILVER SPRING, Md., Feb. 26, 2021 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Nulibry (fosdenopterin) for injection to reduce the risk of death due to Molybdenum Cofactor Deficiency Type A, a rare, genetic, metabolic disorder that typically presents in the first few days of life, causing intractable seizures, brain injury and death. "Today's action marks the first FDA approval for a therapy to treat this devastating disease," said Hylton V. Joffe, M.D., M.M.Sc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA's Center for Drug Evaluation and Research. "The FDA remains committed to facilitating the development and approval of safe and effective therapies for patients affected by rare diseases—an area of critical need."