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Pfizer And Sangamo Start Phase III Study For Hemophilia A Gene Therapy

Giroctocogene Fitelparvovec Sustains Factor VIII Levels With No Bleeds

 
• By Kevin Grogan

The move into Phase III for giroctocogene fitelparvovec comes on the heels of BioMarin's surprise setback when the FDA demanded more proof of durability of its rival hemophilia A gene therapy Roctavian.

New York, July 19, 2016: Corporate lettering on the exterior of the Pfizer headquarters in Manhattan.
Pfizer hoping to lead hemophilia A gene therapy field • Source: Shutterstock

A couple of months after BioMarin Pharmaceutical Inc.’s rival drug Roctavian came unstuck with US regulators, Pfizer Inc. and Sangamo Therapeutics, Inc. have moved their investigational gene therapy for hemophilia A into late-stage studies.

The partners announced that the first participant had been dosed in the Phase III AFFINE trial of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A, the rare genetic disease in which blood does not clot due to a lack of Factor VIII protein

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