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Published: Mar 01, 2021 By Brandon May
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The U.S. Food and Drug Administration (FDA)
has approved BridgeBio Pharma and its affiliate Origin Biosciences’ injectable, rare genetic metabolic disorder therapy NULIBRY™ (fosdenopterin). This approval marks the first treatment indicated for reducing mortality risk in patients with the rare disorder known as molybdenum cofactor deficiency (MoCD) Type A.
An ultra-rare and progressive disease, MoCD Type A affects fewer than 150 patients across the globe. Overall, the median survival following diagnosis is approximately four years. The condition is observed shortly after birth and typically accompanies symptoms such as severe encephalopathy and intractable seizures.
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.
FDA Approves First Treatment for Molybdenum Cofactor Deficiency Type A
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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.