Pfizer announced on Wednesday morning that its gene therapy for hemophilia A met its primary objective in a key clinical trial, and the company plans to bring the data to health regulators for approval in the upcoming months.
The gene therapy, known as giroctocogene fitelparvovec, was non-inferior to routine factor VIII replacement therapy in reducing bleeding rate, meeting the primary endpoint of the trial. The gene therapy showed it was better than factor VIII replacement therapy at reducing bleeding in a superiority analysis, according to Pfizer, and significantly reduced bleeding rates in patients compared to before their gene therapy infusions.
If the therapy is approved, Pfizer would compete with BioMarin, which received approval for its hemophilia A gene therapy Roctavian last year. With slow uptake for Roctavian in its first year, BioMarin said in April it was considering potentially divesting the gene therapy.
Pfizer in April also received approval for a hemophilia B gene therapy Beqvez.
The company said the gene therapy was “generally well tolerated,” with transiently elevated factor VIII levels seen in about half of those who received the gene therapy. Ten patients experienced serious adverse events related to treatment.
Pfizer said it plans to present the full results at a future medical meeting and will bring data to regulators across the world in search of approval.