BioMarin gets go-ahead for Roctavian in the USA

30 June 2023

US biotech BioMarin Pharmaceutical (Nasdaq: BMRN) has won US approval for Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of severe hemophilia A without antibodies to adeno-associated virus serotype 5.

The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the USA. It received approval from the European Medicines Agency in August 2022.

A bumpy road to approval

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