BridgeBio Gets First Drug Approval In Ultra-Rare MoCD Type A
Although small patient base offers modest revenue opportunity, the Nulibry approval enables BridgeBio to get commercial operations underway.
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Since raising $135m in 2017 to address rare, monogenic diseases, BridgeBio has launched or purchased 17 companies and built a pipeline of 30-plus candidates. Scrip checked in as the company nears commercialization.
BridgeBio's hub-and-spoke model is similar to the Roivant and Fortress strategies, but with a focus on drug candidates for genetic diseases in need of a bridge to future funding.
Shares of Alexion Pharmaceuticals rose as much as 6.7% on 24 October on news the FDA deemed the company's synthetic form of cyclic pyranopterin monophosphate (cPMP), known as ALXN1101, a breakthrough therapy – the second product in the firm's pipeline to win the status.