EMA OKs Biomarin’s New Application to Approve Roctavian

EMA OKs Biomarin’s New Application to Approve Roctavian

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EMA OKs Biomarin’s New Application to Approve Roctavian

The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical’s new application seeking approval of its investigational one-time gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. The application was resubmitted to EMA following a request for longer follow-up data from the Phase 3 GENEr8-1 clinical trial (NCT03370913), which is investigating the efficacy of the gene therapy in 134 patients. The same had been requested by the U.S. Food and Drug Administration (FDA)  due to concerns regarding the…

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