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BioWorld - Thursday, February 26, 2026
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Home » Durable goods? Biomarin ‘rox’ in hemophilia A with two-year gene therapy data
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Durable goods? Biomarin ‘rox’ in hemophilia A with two-year gene therapy data

Jan. 10, 2022
By Randy Osborne
Having unveiled more data from the ongoing, global phase III Gener8-1 study with Roctavian (valoctocogene roxaparvovec, also known as valrox), Biomarin Pharmaceutical Inc. remains on track to file a regulatory submission with the FDA in the second quarter of this year for the gene therapy to treat adults with severe hemophilia A. The EMA is already reviewing a marketing authorization application.
BioWorld Clinical Hematologic Gene therapy

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