The Food and Drug Administration approved Pfizer's gene therapy Beqvez (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B, marking a significant milestone as the company's first gene therapy to receive U.S. regulatory clearance. The approval comes with a substantial $3.5 million price tag before insurance and rebates, positioning it among the most expensive drugs in the United States.
Treatment Eligibility and Mechanism
Beqvez is approved for adults with moderate to severe hemophilia B who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or experience repeated serious spontaneous bleeding episodes. Patients must not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid as detected by an FDA-approved test.
The one-time treatment is designed to enable patients to produce factor IX themselves, addressing the underlying cause of hemophilia B. More than 7,000 people in the U.S. live with this debilitating condition, which predominantly affects men and is caused by insufficient levels of factor IX protein that helps blood clot to stop bleeding and seal wounds.
Clinical Advantages Over Standard Care
In late-stage trials, Beqvez demonstrated superiority over the current standard treatment for hemophilia B, which involves administering factor IX protein through regular intravenous infusions multiple times per week or month. This traditional approach is often described as cumbersome and disruptive to patients' daily lives.
"Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues," said Adam Cuker, Director of Penn Medicine's Comprehensive and Hemophilia Thrombosis Program. "A one-time treatment with BEQVEZ has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term."
Market Competition and Pricing Strategy
The gene therapy will compete directly with Australia-based CSL Behring's Hemgenix, a similar treatment that received FDA approval for hemophilia B in 2022. Hemgenix carries a comparable list price of $3.5 million before insurance and rebates. Health experts have noted that high costs and logistical challenges have limited the uptake of Hemgenix and other approved gene therapies for hemophilia.
To address potential concerns about treatment efficacy, Pfizer is offering payers a warranty program for patients who receive Beqvez. The company expects this program to provide "financial protection by insuring against the risk of efficacy failure."
Strategic Significance for Pfizer
The approval represents a crucial development for Pfizer as the company works to regain momentum following the decline of its COVID-19 business. Pfizer is investing heavily in cancer drugs and treatments for other disease areas as part of its business transformation strategy. The company acquired the rights to produce and market Beqvez from Spark Therapeutics in 2014.
Beqvez will be available by prescription to eligible patients this quarter, according to a Pfizer spokesperson. The company is also pursuing FDA approval for its experimental antibody marstacimab to treat both hemophilia A and B, while developing a gene therapy for Duchenne muscular dystrophy.
Gene Therapy Market Evolution
Pfizer joins several companies investing in the rapidly expanding field of gene and cell therapies, which represent one-time, high-cost treatments that target a patient's genetic source or cells to cure or significantly alter disease progression. Health experts anticipate these therapies may eventually replace traditional lifelong treatments used to manage chronic diseases.
