The European Commission (EC) has granted BioMarin Pharmaceutical’s (BioMarin) Roctavian (valoctocogene roxaparvovec) gene therapy a conditional marketing authorisation (CMA) for the treatment of severe haemophilia A.
The authorisation is specifically for the treatment of adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).
People living with haemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and potentially life-threatening bleeds from even modest injuries.
It is estimated that more than 20,000 adults are affected by the condition across more than 70 countries in Europe, the Middle East and Africa, and those with the most severe form of the condition represent approximately 50% of the haemophilia A population.
Roctavian works as a one-time infusion delivering a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued haemophilia prophylaxis, relieving patients of their treatment burden relative to currently available therapies.
The application was supported by a significant body of data from the Roctavian clinical development programme, including two-year outcomes from the global GENEr8-1 phase 3 study which demonstrated stable and durable bleed control. This included a reduction in the mean annualised bleeding rate (ABR) and the mean annualised Factor VIII infusion rate.
Of the 8,000 adults with severe haemophilia A in the 24 countries within BioMarin’s footprint covered by the approval, there are an estimated 3,200 patients who will be indicated for Roctavian, the company said.
Commenting on the approval, Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin, said: “Roctavian approval in Europe is a historic milestone in medicine and is built upon almost four decades of scientific discovery, innovation, and perseverance. We thank the EC for recognising Roctavian’s value as the first gene therapy for haemophilia A, a feat that we believe will transform how healthcare professionals and the patient community think about caring for bleeding disorders.”