Pfizer/Sangamo's gene therapy for hemophilia A was successful in late-stage clinical trials

Pfizer (NYSE: PFE) announced on Wednesday that the experimental gene therapy drug giroctocogene fitelparvovec, jointly developed with Sangamo Therapeutics (NASDAQ: SGMO), has achieved major and major secondary goals in late-stage clinical trials targeting adult hemophilia A.

Citing top-line data from the phase 3 AFFINE trial, the company stated that the diloctocogene fitelparvovec achieved its main goal compared to routine preventive therapy for hemophilia A, a bleeding disorder characterized by a deficiency of blood clotting proteins called blood coagulation factor VIII.

According to PFE, this trial for adult patients with moderate to severe hemophilia A achieved the main goals of non-inferiority and superiority for factor VIII replacement prophylactic therapy based on annual rate converted bleeding rates from week 12 to at least 15 months after infusion.

In terms of safety, the tolerability of girotocogen fitelparvovec was generally good. However, serious adverse events were observed in 20% of study participants, and serious treatment-related adverse events were observed in 13% of them.

However, Pfizer (PFE) said it plans to discuss these results with regulators within the next few months and will also announce additional findings at future healthcare events.