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.
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