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.

Posted In: molybdenum cofactor deficiencyOrphan Drug DesignationBiotechNewsHealth CareFDAGeneral

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