In another shocker, FDA rejects BioMarin’s hemophilia A gene therapy, demanding more data on durability
It turns out that BioMarin’s rivals aren’t the only ones with doubts about the durability of BioMarin’s hemophilia A gene therapy. Regulators at the FDA have some problems with it as well.
In the second major CRL inside of 24 hours, the FDA has slapped down BioMarin’s application for valoctocogene roxaparvovec (valrox) — their top program widely tapped as a cinch at the agency. According to the biotech, they were completely blindsided by a demand to see 2 years of follow-on data from their Phase III trial, rather than the latest update from BioMarin’s investigators.
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