Bleeding rate reduced by 84%, hemophilia A gene therapy phase 3 clinical results listed on NEJM

On March 18, BioMarin Pharmaceutical announced that the results of the Phase 3 clinical trial of the gene therapy valoctocogene roxaparvovec for the treatment of severe hemophilia A were published in the prestigious medical journal The New England Journal of Medicine

Hemophilia A is a rare inherited bleeding disorder caused by a deficiency of factor VIII

Valoctocogene roxaparvovec is a gene therapy that uses an AAV5 viral vector to deliver a transgene expressing Factor VIII

Published study results showed that following a single infusion of valoctocogene roxaparvovec, subjects experienced significantly lower annual bleeding rate (ABR), less frequent factor VIII use, and increased factor VIII activity compared to data from the year prior to enrollment

References:

[1] BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A.

(Original abridged)