Pfizer’s hemophilia gene therapy approved by the FDA

The Food and Drug Administration (FDA) has just greenlighted Pfizer’s gene therapy for a rare genetic bleeding disorder called hemophilia B which causes patients to bleed easily for longer periods of time and also bruise easily. The spontaneous and heavy bleeding caused by the disease can be the aftermath of an injury or surgery.

The disease is caused by the absence or insufficient levels of a protein factor called IX in the blood. Proteins in the blood containing this factor are responsible for sealing wounds and stopping bleeding by helping the blood clot.

The typical course of action in the past for patients suffering from this disease was to seek regular infusions of the blood clotting protein. Pfizer’s one-time gene therapy, Beqvez is meant to offer an alternative by stimulating the production of the protein within the patient’s body, consequently eradicating the need for intravenous infusions of FIX multiple times a week or a month.

The treatment proved its superiority over the standard treatment in a late-stage trial where a single dose of Beqvez was seen to work as well as standard-of-care protein infusions after a year. Beqvez successfully ended bleeding in 60% of the patients it was administered to compared to only the 29% that stand protein infusion was able to stop bleeding.

According to the director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, Adam Cuker, “Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular [factor IX] infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues, Pfizer’s drug has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term.”

The company has stated that it will continue to monitor the drug for the course of the next 15 years to assess the safety and long-term durability of its developed treatment. It was in 2014, through Sparks Therapeutics that Pfixer first acquired the right to market and produce this treatment.

The treatment is expected to become available to patients who fulfill certain requirements by the end of this quarter for a whopping price of $3.5 million before taking into account insurance and other rebates. The drug will be made available to those suffering from severe to moderate hemophilia B through prescription.

The treatment is not just Pfizer’s first gene therapy to be approved in the U.S. But also one of teh company’s successful attempts to push itself out of the decline it fell into after the demand for its COVID business went down last year.

Government data has shown that hemophilia B is more common in men than women and in the U.S. early 4 in every 100,000 U.S. males are affected by the disorder. The number rises to every 12 in 100,000 U.S. males when it comes to hemophilia A.