The US Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) to fidanacogene elaparvovec for the treatment of adults with hemophilia B, Pfizer announced.

“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,” said Chris Boshoff, MD, PhD, chief development officer, oncology and rare disease, Pfizer global product development.

According to phase 3 BENEGENE-2 study safety and efficacy data, the experimental drug was generally safe and well-tolerated as well as more effective than factor IX (FIX) prophylaxis in terms of reducing annualized bleeding rates.

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Moreover, the European marketing authorization application for fidanacogene elaparvovec has been accepted and is under review by the European Medicines Agency. The FDA has set a Prescription Drug User Fee Act goal date in the second quarter of 2024, and it has already granted the experimental drug Breakthrough, Regenerative Medicines Advanced Therapy, and Orphan Drug designations.

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Fidanacogene elaparvovec, an investigational gene therapy containing an artificial adeno-associated virus part and a high-activity variant of the human coagulation FIX gene, aims to enable recipients to produce FIX themselves instead of having to receive regular intravenous infusions of it.


FDA accepts Pfizer’s application for hemophilia B gene therapy fidanacogene elaparvovec. News release. Pfizer Inc.; June 27, 2023.