Factor VIII levels continue to drop in BioMarin’s 3-year update on hemophilia A gene therapy — but wait...
BioMarin dropped their 3-year Phase II update for their hemophilia A gene therapy valrox Tuesday morning, beating their expected deadline in early June and drawing some rapt attention from analysts eagerly awaiting a new set of data on durability. And then, just as the data were sinking in, the biotech threw in a curve ball, offering a snapshot of Phase III data they will be taking to the FDA soon in search of an accelerated approval.
It’s complicated and more than a bit cloudy. But let’s start at the top and work our way down.
As expected, BioMarin concentrated primarily on the big overall drop in bleed rates over the 3-year span, while Wall Street paid more attention to the therapy’s potential for durability, which is all about factor VIII. And those levels continued to fall.
Sign up to read this article for free.
Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.