Pfizer’s gene therapy for a rare genetic blood-clotting disorder called hemophilia A has been successful in late-stage trials, paving the way for potential approval.
Hemophilia A is a lifelong disease caused by a lack of factor VIII protein. Without enough of this protein, blood cannot clot properly, increasing the risk of bleeding episodes. The condition occurs in about 25 per 100,000 male births worldwide.
Pfizer’s one-time treatment significantly reduced the number of annual bleeding episodes in patients with moderately severe to severe hemophilia A after 15 months. This is better than the current standard treatment, which involves routine infusions that replace factor VIII protein.
The study is ongoing, and additional data will be presented at upcoming medical meetings. If approved, Pfizer’s therapy will compete with BioMarin Pharmaceutical’s one-time treatment Roctavian.
Source: https://www.cnbc.com/2024/07/24/pfizers-gene-therapy-for-hemophilia-a-succeeds-in-late-stage-trial.html