BridgeBio Pharma Gets First FDA-Approval For Rare Genetic Metabolic Disorder

BridgeBio Pharma Inc (NASDAQ: BBIO) and affiliate Origin Biosciences Inc have received the FDA approval for Nulibry (fosdenopterin) Injection to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type-A.

• MoCD is a rare metabolic disorder characterized by severe and rapidly progressive neurologic damage caused by the functional loss of sulfite oxidase, 1 of 4 molybdenum-dependent enzymes. 
• The condition is characterized by seizures, feeding difficulties, and encephalopathy (brain damage).
• Nulibry is BridgeBio's first FDA-approved therapeutic, and the company says it is a first-in-class approved cPMP substrate replacement therapy.
• The treatment was reviewed under Priority Review and received FDA Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease Designations.
• Price Action: BBIO shares closed 8.5% higher at 70.68 on Friday.