First Treatment for Molybdenum Cofactor Deficiency Type A approved by FDA
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."
Patients with Molybdenum Cofactor Deficiency Type A experience severe and rapidly progressive neurologic damage including intractable seizures, feeding difficulties and muscle weakness from the accumulation of toxic sulfite metabolites in the central nervous system. Most patients die in early childhood from infections. Before today's approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease.
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