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BioMarin’s Hemophilia Gene Therapy Among Nine Products On Track For EU-Wide Approval

 
• By Vibha Sharma

Four orphan drugs and a new COVID-19 vaccine are among the latest products that the European Medicines Agency says should be approved.

Blood Sample for Factor VIII. Blood coagulation testing. Medical or health concept. coagulation factor test. Antihemophilic factor A test
Hemophilia A Patients Need Frequent Injections To Replace The Missing Factor VIII Blood Clotting Protein • Source: Shutterstock

BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec) is on track to be approved as the first gene therapy in the EU for hemophilia A, after the European Medicines Agency recommended that it be granted a conditional marketing authorization.

Roctavian is intended as a one-time infusion for treating severe hemophilia A in adult patients who do not have factor VIII inhibitors and no antibodies to adeno-associated virus serotype 5

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