FDA Approves First Treatment for Rare Genetic Metabolic Pediatric Disorder

The disorder known as molybdenum cofactor deficiency (MoCD) Type A presents shortly after birth, often with severe encephalopathy and intractable seizures.

The FDA approved the first therapy for an ultra-rare genetic metabolic disorder that affects infants, who don’t normally live past the age of 4.

The disorder is known as molybdenum cofactor deficiency (MoCD) Type A, which presents shortly after birth, often with severe encephalopathy and intractable seizures.

The approval for the injection, fosdenopterin (Nulibry), was granted to BridgeBio Pharma and its affiliate, Origin Biosciences.

A progressive disease, MoCD Type A affects fewer than 150 patients across the globe. It is characterized by an inability to produce cyclic pyranopterin monophosphate (cPMP). The injection replaces missing cPMP in treated patients.

“Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” Hylton V. Joffe, MD, MMSc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, said in a statement. “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.”

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