Food and Drug Administration on Friday announced that the agency had given its approval Pfizertreatment of a scarce gene bleeding disordermaking it company property first gene therapy to obtain authorization in the USA
Agency green delicate the drug, which will be sold under the name Beqvez, for adults with moderate to severe disease hemophilia B who fulfill certain requirements.
A Pfizer spokesperson told CNBC that the drug will be available by prescription to eligible patients this quarter. Its price tag is $3.5 million, not including insurance and other discounts, the spokesman added, making it by far one of the most costly drugs in the US.
More than 7,000 people in the U.S. live with debilitating conditions statewhich, according to an propaganda group. This condition is caused by insufficient levels of a specific protein helps blood clot, stopping bleeding and sealing wounds. Without this protein, called factor IX, patients with hemophilia B bruise easily and bleed more frequently and longer.
Beqvez is a single-use medicine designed to enable patients to produce their own factor IX and prevent and control bleeding. In a late-stage trial, the drug was superior to a drug that was often burdensome standard treatment for hemophilia B, which involves administering protein through the veins several times a week or month.
“Many people with hemophilia B struggle with daily engagement and disruption to their lifestyle [factor IX] infusion, as well as unplanned bleeding episodes that can lead to painful joint damage and mobility problems,” Adam Cuker, director of Penn Medicine’s Comprehensive Thrombosis and Hemophilia Program, said in the Pfizer report release on Friday.
Pfizer’s drug “has the potential to be transformative in the right patients by reducing both medical and treatment burden in the long term,” Cuker added.
The approval is a substantial step for Pfizer, which is trying to regain its footing after a keen decline in its Covid business last year. The company is betting on anticancer drugs and treatments for other diseases to support change the direction of its business.
Pfizer is one of several companies investing in the rapidly growing field of gene and cell therapies – one-time, high-cost treatments that target a patient’s genetic source or cell with the goal of curing or significantly altering the course of a disease. Some health experts expect these therapies to replace classic, lifelong treatments used to treat chronic diseases.
Pfizer gained rights for the production and sale of Beqvez by Spark Therapeutics in 2014.
The company offers a warranty program to payers that covers patients receiving Beqvez, a spokesman told CNBC. Pfizer expects this program to provide “financial protection by providing protection against the risk of efficacy failure,” the release said.
The gene therapy will compete with Australia-based CSL Behring’s therapy Hemgenixsimilar treatment received FDA approval for hemophilia B in 2022. A similar list price for this drug in the US is $3.5 million before insurance and other discounts.
It is worth noting that some health experts claim that this is influenced by, among others: high costs and logistical problems narrow the intake Hemgenix and another approved gene therapy for the more common hemophilia A.
Pfizer is also seeking FDA approval for its experimental antibody, marstacimab, to treat hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic disease that causes gradual muscle weakness.
