BioMarin Pharmaceuticals

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One-time gene therapy for haemophilia A gets FDA green light

3 July 2023

The US Food and Drug Administration (FDA) has approved the first gene therapy for adults with severe haemophilia A. BioMarin’s Roctavian (valoctocogene roxaparvovec-rvox) has been approved for the treatment of adults with severe haemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected […]

Blood in a Petri dish

Haemophilia one-time gene therapy effective for three years

13 January 2023

An investigational one-time gene therapy for the treatment of adults with severe haemophilia A has been proven to be effective for more than three years from treatment.  BioMarin Pharmaceutical revealed the positive results from its ongoing global Phase III GENEr8-1 study of Roctavian (valoctocogene roxaparvovec).   This is the largest and longest global Phase III study […]