Updated: BioMarin’s hemophilia A gene therapy Roctavian wins FDA approval after delay and rejection, priced at $2.9M
The FDA approved the first gene therapy to treat the genetic bleeding disorder hemophilia A on Thursday, capping a turbulent regulatory saga for BioMarin, which will now turn to commercializing the one-time treatment.
Securing the approval for Roctavian was no easy feat. There was a surprise rejection in 2020 and a review extension earlier this year, and now BioMarin has to sell the therapy, which it hopes can grow into a key blockbuster drug that can help double company revenues to $4 billion to $5 billion by the middle of the decade. The drugmaker recorded revenues of about $2.1 billion last year.
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