Pfizer announced on Wednesday that its experimental gene therapy for hemophilia A, a rare genetic blood-clotting disorder, showed positive results in a large late-stage trial. This development could potentially lead to the therapy’s approval and make it Pfizer’s second gene therapy to enter the U.S. market, following the approval of Beqvez in April for hemophilia B.
The gene therapy for hemophilia A is being co-developed by Pfizer and Sangamo Therapeutics. Following the announcement of the trial results, shares of Sangamo Therapeutics surged nearly 40%. Pfizer’s stock also closed up more than 1%. This success highlights Pfizer’s commitment, along with several other drugmakers, to invest in the rapidly growing field of gene and cell therapies.
Gene and cell therapies are one-time, costly treatments that target a patient’s genetic source or cell to cure or significantly alter the course of a disease. Experts in the industry believe that these therapies have the potential to replace traditional lifelong treatments for managing chronic conditions. This could be a game-changer for patients with hemophilia A, a lifelong disease caused by a lack of the blood-clotting protein factor VIII.
Without enough factor VIII protein, the blood cannot clot properly, leading to an increased risk of spontaneous bleeding and severe bleeding after surgery. Pfizer’s one-time treatment demonstrated significant reduction in the number of annual bleeding episodes in patients with moderately severe to severe hemophilia A. The therapy also outperformed the current standard treatment, which involves routine infusions to replace the factor VIII protein.
Dr. Andrew Leavitt, the lead investigator of the trial, emphasized the physical and emotional impact of frequent IV infusions or injections for people living with hemophilia A. The positive results from Pfizer’s study offer hope for a more effective and convenient treatment option for these patients.
While Pfizer’s therapy shows promise, it will face competition from BioMarin Pharmaceutical’s one-time treatment, Roctavian. BioMarin’s therapy was approved in the U.S. last year but has had a slow rollout, raising questions about the potential uptake of Pfizer’s drug in the market. BioMarin is reportedly considering divesting its hemophilia A therapy, which comes with a hefty price tag of $2.9 million.
Pfizer’s gene therapy for hemophilia A is still undergoing further study, and additional data will be presented at upcoming medical meetings. If approved, this therapy has the potential to revolutionize the treatment landscape for patients with hemophilia A, offering them a more convenient and effective alternative to current lifelong treatments.
