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BridgeBio Gets First Drug Approval In Ultra-Rare MoCD Type A

 
• By Joseph Haas

Although small patient base offers modest revenue opportunity, the Nulibry approval enables BridgeBio to get commercial operations underway.

FDA approved background, 3D rendering, blue street sign
Nulibry for MoCD type A is BridgeBio's first approved drug

With a projected patient base of about 150 infants globally, BridgeBio Pharma, Inc. believes its newly approved Nulibry (fosdenopterin) will treat the smallest patient population for which a drug was solely created and approved. Nulibry, a recombinant form of cyclic pyranopterin monophosphate (cPMP), received US Food and Drug Administration (FDA) approval on 26 February to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A.

MoCD type A is an ultra-rare and progressive disease that usually manifests within days of birth, causing severe encephalopathy and...

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