FDA Accepts Pfizer’s Application for Hemophilia B Gene Therapy Fidanacogene Elaparvovec

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FDA Accepts Pfizer’s Application for Hemophilia B Gene Therapy Fidanacogene Elaparvovec

Source – Pfizer

On 28 June 2023, Pfizer announced  that the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have accepted the Biologics License Application (BLA) and marketing authorization application (MAA), respectively, for fidanacogene elaparvovec, a groundbreaking gene therapy developed for the treatment of hemophilia B in adults.

Fidanacogene elaparvovec is an innovative gene therapy that utilizes a modified adeno-associated virus (AAV) capsid and a high-activity variant of the human coagulation Factor IX (FIX) gene. The aim of this therapy is to enable individuals with hemophilia B to produce their own FIX through a one-time treatment, eliminating the need for regular intravenous infusions of FIX, which is the current standard of care.

“Gene therapy marks a new era of scientific advancement, and if approved, we believe fidanacogene elaparvovec has the potential to transform the lives of people living with hemophilia B who are eligible for treatment. We look forward to continuing to work with global regulatory authorities to bring this innovative potential treatment to patients as quickly as possible. Patients are at the center of our legacy of innovation in hemophilia. Despite significant progress in their treatment, those living with hemophilia continue to experience disruption to daily life and need new options.”

– Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development

The applications for fidanacogene elaparvovec are supported by compelling efficacy and safety data from the Phase III BENEGENE-II study4. As previously reported, this study successfully met its primary endpoint by demonstrating non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds after fidanacogene elaparvovec infusion compared to prophylaxis regimen with FIX, which is administered as part of usual care. The therapy exhibited a generally well-tolerated safety profile consistent with the results from Phase I/II trials.

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date for the second quarter of 2024 to complete the review process. Fidanacogene elaparvovec has received Breakthrough, Regenerative Medicines Advanced Therapy (RMAT), and orphan drug designations from the FDA.

Pfizer is actively conducting three Phase III programs investigating gene therapy for high unmet medical needs in hemophilia B, hemophilia A, and Duchenne muscular dystrophy. Additionally, a Phase III trial is underway to evaluate marstacimab, a novel anti-tissue factor pathway inhibitor (anti-TFPI), for the treatment of individuals with hemophilia A and B, with or without inhibitors.

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