US approves gene therapy treatment for hemophilia
The US Food and Drug Administration (FDA) has approved Pfizer’s gene therapy Beqvez, marking a significant advancement in the treatment of hemophilia B, a rare inherited blood-clotting disorder. The approval follows a clinical trial that demonstrated the treatment’s effectiveness in reducing bleeding among adults with moderate to severe hemophilia B, a form of the disease primarily affecting males.
Beqvez is administered as a single intravenous infusion and has shown promising results in a trial involving 45 participants. The study found that the therapy significantly reduced bleeding episodes compared to the current standard of care, which involves regular infusions of the clotting protein factor IX (FIX). This conventional treatment can be cumbersome, requiring infusions several times a week, posing a significant burden on patients’ lifestyles.
Adam Cuker, the director of the University of Pennsylvania’s Comprehensive Hemophilia and Thrombosis Program, explained that many people with hemophilia B face challenges due to the frequency of FIX infusions and the associated disruptions to their lives. Additionally, these patients often experience spontaneous bleeding episodes that can lead to painful joint damage and reduced mobility. He emphasized that Beqvez’s one-time treatment could be transformative for suitable patients by reducing both the medical and treatment burdens over the long term.
Although the gene therapy was generally well-tolerated in clinical trials, some patients experienced elevated liver enzymes, indicating potential liver inflammation, albeit without visible symptoms. As a precaution, patients are advised to avoid alcohol for up to a year following treatment to prevent liver damage. Pfizer plans to monitor patients for up to 15 years to gather long-term data on the therapy’s safety and effectiveness.
Beqvez targets hemophilia B by introducing a modified virus that delivers a functional copy of the factor IX gene to liver cells, prompting them to produce the clotting protein that the patient otherwise lacks. This approach offers a significant improvement over traditional treatments. While the list price of the therapy is set at $3.5 million, Pfizer indicates that the cost for insured patients would likely be lower. Despite the hefty price tag, the company argues that Beqvez’s one-time treatment could be more cost-effective in the long run compared to ongoing traditional treatments, which can cost between $600,000 and $1.1 million annually.
This approval by the FDA follows the gene therapy’s earlier approval in Canada and its current review by the European Medicines Agency. As Beqvez enters the market, it has the potential to change the lives of thousands of people living with hemophilia B, offering them a new path to manage their condition with reduced medical intervention and improved quality of life.
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