The Food and Drug Administration recently approved Pfizer’s gene therapy for a rare genetic bleeding disorder called hemophilia B. The treatment, called Beqvez, is intended for adults with moderate to severe hemophilia B who meet specific criteria. This one-time treatment aims to enable patients to produce factor IX, a protein necessary for blood clotting, to help prevent and control bleeding. The approval of Beqvez marks Pfizer’s entry into the gene therapy market in the U.S., with a price tag of $3.5 million before insurance and rebates. Hemophilia B affects more than 7,000 people in the U.S., predominantly men, causing them to bruise easily and bleed for extended periods without the proper clotting protein.
A late-stage trial found Beqvez to be superior to the standard treatment for hemophilia B, which involves regular infusions of factor IX through the veins. This cumbersome routine can disrupt patients’ lives and lead to painful joint damage and mobility issues due to spontaneous bleeding episodes. Dr. Adam Cuker, director of Penn’s Comprehensive and Hemophilia Thrombosis Program, highlighted the potential of Pfizer’s gene therapy to reduce the long-term medical and treatment burden on appropriate patients. Pfizer’s entry into the gene therapy market comes as the company seeks to revamp its business following the decline of its Covid-related products. By focusing on cancer drugs and treatments for various diseases, Pfizer aims to drive growth and innovation in the healthcare industry.
Pfizer secured the rights to produce and market Beqvez from Spark Therapeutics in 2014 and is offering payers a warranty program to provide financial protections in case of efficacy failure. The gene therapy will compete with CSL Behring’s Hemgenix, another treatment for hemophilia B that was approved by the FDA in 2022. Both Beqvez and Hemgenix have a list price of $3.5 million in the U.S., but there have been concerns about the high costs and logistical challenges associated with gene therapies that have limited their uptake. Despite these challenges, Pfizer is also seeking FDA approval for its experimental antibody marstacimab to treat hemophilia A and B, as well as developing a gene therapy for Duchenne muscular dystrophy, a genetic disorder that causes muscle weakness over time.
Gene and cell therapies are one-time, high-cost treatments that target the genetic source or cells of a disease to potentially cure or significantly alter its course. Health experts believe that these innovative therapies have the potential to replace traditional lifelong treatments for chronic diseases. By investing in gene and cell therapies, Pfizer and other companies are at the forefront of a rapidly growing field that offers new hope for patients with rare genetic disorders. The approval of Beqvez marks a significant milestone for Pfizer’s gene therapy portfolio and underscores the company’s commitment to advancing cutting-edge treatments for patients in need.
In conclusion, the FDA approval of Pfizer’s gene therapy for hemophilia B represents a breakthrough in the treatment of rare genetic disorders. Beqvez offers potential benefits for patients with moderate to severe hemophilia B by reducing the medical and treatment burden over the long term. Despite the high costs and logistical challenges associated with gene therapies, Pfizer’s entry into this market highlights the company’s commitment to innovation and improving patient outcomes. With the successful development of Beqvez and ongoing research into other gene therapies, Pfizer is poised to make a significant impact in the healthcare industry and provide hope for individuals living with rare genetic conditions.
