A request for more information from the US Food and Drug Administration has once again pushed back the date when BioMarin Pharmaceutical Inc. can refile its biologics license application (BLA) for the gene therapy Roctavian (valoctocogene roxaparvovec) in hemophilia A, delaying the planned resubmission by up to three months. However, the company is better prepared to hit the market in the US as well as Europe than it was the last time around.
BioMarin released updated data on 31 May from its Phase I/II study of Roctavian showing six-year follow-up data for patients receiving the 6e13 vg/kg – the dose used in the pivotal Phase III GENEr8-1 study – and five-year data for the 4e13 vg/kg cohorts. The company also said that the FDA had requested additional information and analyses of data to include in its BLA package
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