Cure for hemophilia A still effective after three years

9 January 2023

A gene therapy for hemophilia A has shown positive results after three years of follow up in a major late-stage clinical trial.

Developed by Californian genetics specialist BioMarin Pharmaceutical (Nasdaq: BMRN), Roctavian (valoctocogene roxaparvovec) is a one-time treatment for adults with severe hemophilia A.

Data from the ongoing GENEr8-1 study of the candidate show that the average bleed rate was reduced by 80% from baseline, while usage of Factor VIII was reduced by 94% in the third year.

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