Pfizer’s Hemophilia A Gene Therapy Superior To Prophylaxis In Phase III

The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.

A Phase III trial of Pfizer's hemophilia A gene therapy was successful • Source: Shutterstock

Pfizer Inc. appears closer to launching a gene therapy for hemophilia A, based on the positive results of the Phase III AFFINE study testing giroctocogene fitelparvovec for the treatment of moderately severe to severe hemophilia A versus prophylaxis.

The company announced the positive data on 24 July, reporting the AFFINE study met its primary endpoint of non-inferiority, as well as superiority, on total annualized bleeding rate (ABR) from week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII replacement prophylaxis treatment

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