Pfizer’s First Of Its Kind Gene Therapy Shines In Phase 3 Trials For Treatment of Hemophilia A

1. Giroctocogene fitelparvovec, a novel gene therapy, demonstrated superiority to routine therapy in treating hemophilia A.
2. Giroctocogene fitelparvovec resulted in a statistically significant reduction in bleeding events, sustained improvement in factor VIII activity, and was well tolerated by participants.

The Latest

In an open-label, single-arm, phase 3 clinical trial, researchers at Pfizer investigated the safety and efficacy of giroctocogene fitelparvovec (GF), a first-of-its-kind gene therapy, for the treatment of moderate to severe Hemophilia A in adult male participants. Results were compared to routine prophylactic factor VIII replacement therapy. The study found that GF was not only non-inferior but superior to routine prophylaxis. GF showed a statistically significant reduction in bleeding events, and sustained factor VIII activity, and was well tolerated by participants.

Physician’s Perspective

Hemophilias are a group of bleeding disorders. There are three types of hemophilia, each defined by a deficiency in a specific molecule involved in the clotting process. Hemophilia A is a deficiency in factor VIII and is typically genetically inherited, specifically X-linked which means males are classically affected. The condition can be diagnosed based on history and a measurement of factor VIII levels. Patients with hemophilia are typically only treated when they are symptomatic or anticipating surgery. Treatments include replacement of the clotting factors, antifibrinolytics to prevent the breakdown of clots, and desmopressin to increase factor VIII concentrations. There is presently no curative treatment for the disorder. Individuals with moderate to severe hemophilia require routine prophylactic therapy. Pfizer’s GF could be a game-changing treatment in the hemophilia landscape by providing prolonged improvement in clotting factor levels.

Molecular Target of Therapy

GF is a genetic therapy that contains a modified human coagulation factor VIII gene. It is administered via intravenous infusion. The functional factor VIII gene is delivered to liver cells and incorporated into the cell nucleus. The cells can then use this gene to create endogenous factor VIII.

Company History

Pfizer is an American multinational pharmaceutical company. The company is known for its substantial contributions to the pharmaceutical landscape. Its successes include sildenafil (Viagra) for erectile dysfunction, apixaban to prevent blood clots, and the COVID-19 vaccine developed in partnership with BioNTech. Pfizer continues to research and develop numerous pharmaceuticals. It currently has 5 candidates in the registration phase, one of which is a monoclonal antibody (marstacimab) also for treating hemophilia.

Further reading: https://www.pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-topline-results-phase-3-study

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