How durable is durable? BioMarin gears up to bring hemophilia A gene therapy to FDA again with 2-year data
A year and a half after a stunning rejection, BioMarin says it has the durability data the FDA previously requested, paving the way for a new BLA in the second quarter of this year.
Touting “stable and durable” bleed control in what it calls the largest ever global study of a gene therapy involving patients with severe hemophilia A, BioMarin notes its drug, valoctocogene roxaparvovec (better known as valrox or Roctavian), met all primary and secondary efficacy endpoints at the two-year analysis.
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