FDA green lights first treatment for molybdenum cofactor deficiency Type A

The US Food and Drug Administration on Friday 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.

The FDA granted the approval of Nulibry to Origin Biosciences, an affiliate of BridgeBio Pharma (Nasdaq: BBIO), whose shares gained 8.5% to $70.68 on the news. The FDA accepted the New Drug Application (NDA) for fosdenopterin (previously BBP-870/ORGN001) in September 2020.

“Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Dr Hylton Joffe, 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,” he noted.