BioMarin returns to FDA with a new pitch for hemophilia gene therapy — and a promise to get to the bottom of durability
Two years ago, the FDA slapped a surprise rejection on BioMarin’s BLA for its gene therapy for hemophilia A, demanding two-year data from a Phase III trial to answer questions about durability.
BioMarin is now back with a resubmission — with the data that the FDA asked for and a promise for even more.
The new BLA filing comes a month after the European Commission granted conditional approval to valoctocogene roxaparvovec, more colloquially known as valrox, under the brand name Roctavian.
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