Pfizer’s First Gene Therapy Approved by FDA for Rare Genetic Bleeding Disorder
The Food and Drug Administration (FDA) announced on Friday that it has approved Pfizer’s treatment for a rare genetic bleeding disorder, making it the company’s first gene therapy to receive approval in the United States. The drug, known as Beqvez, has been approved for use in adults with moderately severe hemophilia B who meet certain criteria.
Hemophilia B is a debilitating condition that affects more than 7,000 Americans. It is caused by insufficient levels of a protein called factor IX, which helps blood clot to stop bleeding and seal wounds. Without this protein, patients with hemophilia B often experience easy bruising and frequent, prolonged bleeding.
Pfizer’s Beqvez is a one-time treatment that aims to help patients produce factor IX on their own, as well as prevent and control bleeding. It has been shown to be superior to the traditional treatment for hemophilia B, which involves injecting the protein into the veins multiple times a week or month.
Adam Cuker, director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, believes that Pfizer’s drug has the potential to transform the lives of appropriate patients by reducing both the medical burden and the burden of treatment over the long term. Many individuals with hemophilia B struggle with the commitment and lifestyle disruptions associated with regular factor IX infusions, as well as spontaneous bleeding episodes that can lead to painful joint damage and mobility problems.
However, the price tag on this new drug is hefty at $3.5 million before insurance and other rebates, making it one of the most expensive drugs available in the United States. Pfizer is offering a warranty program to provide financial protection for payers covering patients who receive Beqvez, ensuring against the risk of efficacy failures.
This approval is a significant milestone for Pfizer as it tries to recover from the decline of its Covid business last year. The company is heavily investing in cancer drugs and treatments for other diseases to turn around its business. Pfizer is not alone in its focus on gene and cell therapies, as many companies are investing in this rapidly growing field. These one-time high-cost treatments target the genetic source or cell of a patient to cure or significantly alter the course of a disease. Health experts believe that these therapies may replace traditional lifelong treatments for chronic illnesses.
Pfizer acquired the rights to produce and market Beqvez from Spark Therapeutics in 2014. Hemgenix, a similar treatment for hemophilia B, was approved by the FDA in 2022 and will compete with Pfizer’s gene therapy. It is developed by Australia-based CSL Behring and has a similar price tag of $3.5 million before insurance and other rebates in the United States.
Hemgenix has faced challenges in becoming widely available for the more common hemophilia A disease due to high costs and logistical issues. However, CSL Behring is seeking FDA approval for its experimental antibody, marstacimab, which is being developed for the treatment of both hemophilia A and B. The company is also working on a genetic therapy for Duchenne muscular dystrophy, a condition that causes gradual muscle weakness over time.
In conclusion, Pfizer’s approval of Beqvez marks an important milestone for gene therapy in the United States. While the drug offers potential benefits for patients with hemophilia B, its high cost raises concerns about accessibility. Nevertheless, the field of gene and cell therapies continues to advance, promising new possibilities for the treatment of various diseases.