The European Commission has granted conditional marketing authorization for Pfizer’s DURVEQTIX® (fidanacogene elaparvovec), a gene therapy for treating severe and moderately severe hemophilia B in adults without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74. This approval marks a significant advancement in hemophilia B treatment, potentially transforming the standard of care .
Many patients are offered a one-time gene therapy that enables the production of factor IX (FIX), thus reducing or eliminating the need for frequent intravenous FIX infusions, which are currently required to manage the disease. Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting due to a deficiency in FIX, causing individuals to bleed more frequently and for longer periods than others.
The current standard of care for hemophilia B involves regular prophylactic infusions of FIX replacement therapy, which temporarily replace or supplement low levels of blood-clotting factor. Despite these prophylactic treatments and regular intravenous infusions, many people living with moderate to severe hemophilia B are at risk of spontaneous bleeding episodes. This places a significant burden not only on patients but also on healthcare systems’ budgets and resource utilization.
Impact on Hemophilia B Care in the European Union
Alexandre de Germay, Pfizer’s Chief International Commercial Officer, highlighted the transformative potential of DURVEQTIX for hemophilia B care in the European Union. The gene therapy could significantly reduce or eliminate bleeding episodes for appropriate patients, addressing the substantial medical and treatment burden associated with the current standard of care. Hemophilia B is characterized by a deficiency in FIX, leading to frequent and prolonged bleeding episodes. This deficiency necessitates regular and often cumbersome treatment regimens that can strain patients and healthcare systems alike.
The BENEGENE-2 study, which played a pivotal role in the approval process, enrolled 45 participants with moderately severe to severe hemophilia B. This open-label, single-arm study aimed to evaluate the efficacy and safety of DURVEQTIX by comparing the annualized bleeding rate (ABR) of participants treated with the gene therapy against their ABR during a lead-in period on a FIX prophylaxis regimen.
The study met its primary efficacy endpoint, demonstrating a statistically significant decrease in the model-based ABR from 4.50 during the lead-in period to 1.44 at the 15-month follow-up period, representing a 68% reduction. Furthermore, DURVEQTIX eliminated bleeds in 62.2% of patients. The safety profile of DURVEQTIX was consistent with previous clinical trials, with the most common adverse reaction being an increase in liver enzymes, which was managed with corticosteroids.
This conditional marketing authorization is valid in all 27 EU member states, as well as in Iceland, Liechtenstein, and Norway. The approval follows recent regulatory approvals by the U.S. Food and Drug Administration (FDA) and Health Canada, where the therapy is marketed as BEQVEZ™. This broad regulatory acceptance underscores the potential global impact of DURVEQTIX in transforming hemophilia B care.
Pfizer’s Commitment to Hemophilia Care
Pfizer’s approval of DURVEQTIX builds on its more than 40-year commitment to delivering breakthrough solutions for people living with hemophilia. In addition to DURVEQTIX, Pfizer is advancing a Phase 3 program investigating a gene therapy in hemophilia A, known as giroctocogene fitelparvovec, and a Phase 3 trial for marstacimab, a novel anti-tissue factor pathway inhibitor for hemophilia A and B patients with and without inhibitors. Pfizer’s dedication to pushing the boundaries of hemophilia treatment aims to improve patient outcomes and quality of life significantly.
These advancements reflect Pfizer’s strategic efforts to address the unmet needs of hemophilia patients through innovative therapies. The company’s robust hemophilia pipeline includes potential treatments that leverage cutting-edge gene therapy techniques to offer long-term, sustainable solutions to bleeding disorders. This commitment to research and development is crucial in a landscape where the standard of care often involves lifelong, burdensome treatment regimens.
The conditional marketing authorization of DURVEQTIX by the European Commission represents a pivotal moment in hemophilia B treatment. By enabling patients to produce FIX internally through a one-time gene therapy, DURVEQTIX offers a potentially transformative alternative to the regular infusions required by current therapies. This innovation not only promises to improve the quality of life for patients but also to alleviate the treatment burden and associated healthcare costs.
Pfizer continues to collaborate with healthcare providers, governments, and local communities to support and expand access to innovative therapies. This approach aligns with Pfizer’s long-standing mission to bring therapies to people that extend and significantly improve their lives, setting the standard for quality, safety, and value in healthcare.
Resource: Pfizer, July 25, 2024
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