Pfizer, Inc. has announced that they are ceasing global development and commercialization of Beqvez™, the company’s hemophilia B gene therapy product.
Beqvez was designed with bioengineered adeno-associated virus (AAVs) vectors to introduce a working copy of the factor IX (FIX) gene to generate therapeutic levels of clotting FIX. It received approval from the U.S. Food and Drug Administration less than a year ago, in the spring of 2024.
According to a recent Fierce Pharma news post, several reasons led to the discontinuation, including “limited interest” from patients and doctors toward hemophilia gene therapies to date, a Pfizer spokesperson told Fierce Pharma in a statement.
People with hemophilia, their families, and healthcare providers must weigh several considerations and unknowns when deciding on whether to embark on a one-time treatment such as gene therapy. These include questions about long-term safety and efficacy, cost, and the potential promise of other advanced therapies.
Supplemental NBDF Resources-
Sources: Reuters, February 20, 2025, and Fierce Pharma, February 21, 2025
