Biomarin's hemophilia drug meets all endpoints in phase III study

Biomarin has released results from a phase III trial of gene therapy valoctocogene roxaparvovec against severe hemophilia A.

On Sunday, Biotech company Biomarin publicized results from the largest study of a gene therapy hemophilia A treatment ever, at least according to the company itself.

A total of 134 patients have participated in the late-stage trial with the drug candidate valoctocogene roxaparvovec, which was rejected by the US Food and Drug Administration in August 2020 and is currently being evaluated by the European Medicines Agency (EMA).

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