Pfizer’s Gene Therapy Approved by FDA to Treat Rare Blood Disorder Hemophilia B

The Food and Drug Administration (FDA) has approved Pfizer’s first-ever gene therapy in the US, which will be used to treat adults with moderate to severe hemophilia B who meet certain criteria. The drug, called Beqvez, is designed to enable patients to produce a protein that helps blood form clots and prevent bleeding associated with this rare genetic disorder caused by insufficient levels of factor IX. More than 7,000 people in the US are living with hemophilia B, which leads to easy bruising and prolonged periods of excessive bleeding due to low amounts of factor IX protein.
In a late-stage trial, Pfizer’s gene therapy was found to be superior to standard treatments for haemophilia B that require repeated infusions via veins every few weeks or months because it can cause painful joint damage and mobility issues over time. “Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular [factor IX] infusions, as well as spontaneous bleeding episodes,” said Dr Adam Cuker from Penn’s Comprehensive Hemophilia Thrombosis Program in a Pfizer release on Friday.
The approval is significant for Pfizer which has been trying to regain its footing following the rapid decline of its Covid business last year, and it hopes that gene therapies will help turn around its fortunes by investing heavily in cancer drugs and treatments for other disease areas. The company gained rights from Spark Therapeutics in 2014 to produce and market Beqvez but has not disclosed the price yet.
The rapid growth of cell and gene therapy is one-time, high-cost treatments that target a patient’s genetic source or cells instead of using long-term traditional therapies for chronic diseases such as cancer drugs or insulin injections to manage diabetes. However, some health experts have noted logistical issues and high costs may limit the uptake of these gene therapy products approved by regulators recently. Hemogenix is an example – a similar treatment produced by CSL Behring that won FDA approval for hemophilia B last year has a list price in the US before insurance rebates or discounts of $3.5 million (£2,796,480).