Pfizer Gets FDA Approval For Rare Inherited Bleeding Disorder Gene Therapy

(CTN News) – In announcing its approval on Friday, the Food and Drug Administration said it had 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.

It was approved by the FDA for use in adults with moderately severe hemophilia B who meet certain criteria. The drug will be marketed as Beqvez.

In this quarter, eligible patients will be able to obtain the treatment by prescription, according to a Pfizer spokesperson. According to the spokesperson, the price tag on this 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.

According to an advocacy group, more than 7,000 Americans live with this debilitating condition, which typically affects men. It is caused by insufficient levels of a protein that helps blood clot in order to stop bleeding and seal wounds. A patient with hemophilia B who lacks this protein, called factor IX, will bruise easily and bleed more frequently and for a longer period of time.

A one-time treatment known as Beqvez is intended to help patients produce factor IX on their own as well as prevent and control bleeding. The drug was shown to be superior to the traditional treatment for hemophilia B, which involves injecting the protein into the veins multiple times a week or a month.

In the Pfizer release on Friday, Adam Cuker, director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, stated, “Many individuals with hemophilia B struggle with the commitment and lifestyle disruptions associated with regular [factor IX] infusions, as well as spontaneous bleeding episodes, which can result in painful joint damage and mobility problems.”

According to Cuker, 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.”

This approval represents an important step for Pfizer, which is trying to regain its footing after the rapid decline of its Covid business last year. The company is betting heavily on cancer drugs and treatments for other diseases in order to turn around its business.

Pfizer is one of many companies investing in the rapidly growing field of gene and cell therapies – one-time high-cost treatments that target the genetic source or cell of a patient in order to cure or significantly alter the course of a disease. According to some health experts, these therapies may replace traditional lifelong treatments that are used to manage chronic illnesses.

Spark Therapeutics sold its rights to Pfizer in 2014 for production and marketing of Beqvez.

According to a spokesperson for the company, a warranty program is available for payers to cover patients who receive Beqvez. Specifically, Pfizer expects that the program will provide “financial protection” by ensuring against the risk of efficacy failures.

Hemgenix, a similar treatment approved by the FDA in 2022 for hemophilia B, will compete with the gene therapy developed by Australia-based CSL Behring. According to the company, the price of the drug in the U.S. is similar at $3.5 million before insurance and other rebates.

In particular, some health experts have stated that high costs and logistical issues have prevented Hemgenix from becoming more widely available for the more common hemophilia A disease.

It is also seeking approval from the FDA for its experimental antibody, marstacimab, which is being developed for the treatment of hemophilia A and B. Additionally, the company is developing a genetic therapy for Duchenne muscular dystrophy, a condition that causes muscles to weaken gradually over time.

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