BioMarin spells out a troubling slide in Factor VIII levels for its big blockbuster contender valrox
We already knew from BioMarin’s statement a few days ago that their gene therapy for hemophilia A continued to protect patients against the bleeding episodes that can threaten their lives. Now, 4 years in, we’re getting the hard data on durability for valrox — and here the biotech faces considerable potential trouble.
As Stifel’s Paul Matteis noted recently, at the end of the first, second and third years, post-infusion, mean Factor VIII activity level of the high dose 6e13 vg/kg cohort was 64.3, 36.4 and 32.7 IU/dL respectively as measured by the CS assay.
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