The Food and Drug Administration has approved Pfizer’s treatment for a rare genetic bleeding disorder, making it the company’s first gene therapy to receive clearance in the U.S. The drug, called Beqvez, is intended for adults with moderate to severe hemophilia B. More than 7,000 people in the U.S. are affected by this condition, which is caused by a lack of a specific protein that helps blood clot. Beqvez is a one-time treatment that enables patients to produce the needed factor IX protein themselves to prevent and control bleeding, offering a potential alternative to the cumbersome standard treatment of administering protein infusions multiple times a week.
Pfizer’s Beqvez has been found to be superior to the standard treatment for hemophilia B in a late-stage trial, by reducing the need for frequent infusions and lowering the risk of spontaneous bleeding episodes that can cause painful joint damage and mobility issues. The approval of Beqvez is a significant milestone for Pfizer as it seeks to expand its presence in the gene and cell therapy market and recover from the impact of the decline in its COVID-related business. The company is investing in cancer drugs and treatments for various disease areas to drive growth. Gene and cell therapies are seen as a promising new approach to treating diseases by targeting a patient’s genetic source or cells to cure or change the course of a condition, potentially replacing traditional lifelong treatments for chronic diseases.
Pfizer acquired the rights to Beqvez from Spark Therapeutics in 2014 and is offering a warranty program to provide financial protection to patients receiving the gene therapy. The high cost of Beqvez, priced at $3.5 million in the U.S. before rebates, puts it among the most expensive drugs on the market. However, Pfizer aims to mitigate the financial burden on patients and payers by insuring against the risk of efficacy failure. Beqvez will compete with CSL Behring’s Hemgenix, a similar treatment for hemophilia B that received FDA approval in 2022. Despite the potential benefits of gene therapies, high costs and logistical challenges have limited the uptake of such treatments for hemophilia and other conditions.
In addition to Beqvez, Pfizer is seeking FDA approval for its experimental antibody, marstacimab, to treat both hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic disorder that causes progressive weakening of muscles. With the increasing interest and investment in gene and cell therapies, Pfizer is strategically positioning itself to capitalize on the growth potential of this rapidly evolving field of medicine. By expanding its portfolio of innovative treatments, Pfizer aims to enhance its competitive position in the healthcare industry and drive long-term value for patients and shareholders alike.
Overall, the approval of Pfizer’s Beqvez gene therapy for hemophilia B represents a significant advancement in the treatment of this rare genetic bleeding disorder. By providing a one-time treatment option that enables patients to produce the necessary clotting protein themselves, Beqvez offers the potential to reduce the burden of frequent infusions and prevent bleeding episodes in individuals with hemophilia B. Pfizer’s entry into the gene therapy market reflects its strategic focus on developing innovative treatments for a range of diseases, including cancer, hemophilia, and Duchenne muscular dystrophy. Despite the high cost of gene therapies, the increasing investment in this area holds promise for transforming the treatment landscape and improving outcomes for patients with genetic disorders.
