Gene Therapy Reduces Bleeding in Hemophilia A Patients: Late-Stage Trial Results
Reuters
July 24, 2024
Pfizer's gene therapy for hemophilia A significantly reduced the number of annual bleeding episodes in patients with the disorder in a late-stage study and performed better than the current standard treatment, according to the company.
This data brings Pfizer closer to securing U.S. regulatory approval for a second gene therapy to treat hemophilia.
Pfizer previously received FDA approval for Beqvez, a one-time gene therapy for hemophilia B, a less common type of the disorder, in April.
If approved, Pfizer's hemophilia A gene therapy will compete with BioMarin Pharmaceutical's one-time treatment Roctavian, which was approved in the U.S. last year.
Currently, the standard treatment for hemophilia A patients involves routine replacement of the clotting protein Factor VIII.
Pfizer reported that its gene therapy for hemophilia A not only met the primary endpoint of demonstrating non-inferiority compared to Factor VIII replacement therapy in reducing the annual bleeding rate but also showed superiority to the current standard treatment.
Data from the 75-patient trial indicated that 84% of patients who received Pfizer's gene therapy had Factor VIII levels above 5% at 15 months post-infusion.
The company will present additional data at upcoming medical meetings. Pfizer is co-developing the hemophilia A gene therapy with Sangamo Therapeutics, from whom it licensed the therapy.
Stay up to date with the latest clinical headlines.
Thank you for signing up for the Daily News alerts. You will begin receiving them shortly.