Pfizer's Gene Therapy Fidanacogene Elaparvovec Receives FDA Priority Review for Hemophilia B

On June 27, Pfizer announced that the FDA had accepted and granted Priority Review to the Biologics License Application (BLA) for its gene therapy fidanacogene elaparvovec (SPK-9001) for the treatment of hemophilia B, with an FDA action date (FDUFA) set for Q2 2024. Meanwhile, the Marketing Authorization Application (MAA) for this product has also been accepted by the European Medicines Agency (EMA).

Fidanacogene elaparvovec is a gene therapy developed by Spark Therapeutics (acquired by Roche) that targets coagulation factor IX (FIX). It contains a bioengineered adeno-associated virus (AAV) capsid (protein shell) and a highly active variant of the human FIX gene. In December 2014, Pfizer partnered with Spark to secure global rights to the product in a deal worth $280 million.

This BLA is primarily based on the positive data from the Phase III BENEGENE-2 study (n=45). The results showed that within 12 months of receiving a single dose of fidanacogene elaparvovec (5e11 vg/kg), the annualized bleeding rate (ABR) in patients significantly decreased compared to the standard prophylaxis group (receiving FIX drugs as preventive replacement therapy) (1.3 vs. 4.43, P<0.0001), meeting both non-inferiority and superiority criteria.

Pfizer has laid out six products in the hemophilia field. Apart from the three marketed coagulation factor drugs, the remaining three (fidanacogene elaparvovec, giroctocogene fitelparvovec, and marstacimab) are all in the late-stage research phase. Giroctocogene fitelparvovec is a gene therapy for Hemophilia A, with its pivotal Phase III clinical trial expected to be completed by July 2024. Marstacimab is a monoclonal antibody targeting Tissue Factor Pathway Inhibitor (TFPI) for Hemophilia A and B, which completed a pivotal clinical trial in May this year (see: ).

Hemophilia B is a hereditary hemorrhagic disease caused by congenital FIX deficiency, also known as congenital FIX deficiency or Christmas disease. According to statistics from the World Federation of Hemophilia, there were over 38,000 patients with hemophilia B globally in 2021. Currently, 13 drugs have been approved worldwide for the treatment of hemophilia B, 11 of which are coagulation factor drugs. Concizumab, a TFPI monoclonal antibody developed by Novo Nordisk, has been approved in Canada for the treatment of hemophilia B; however, the product failed to receive FDA approval last month (see: ). Etranacogene dezaparvovec, a gene therapy developed by CSL/uniQure, was approved for marketing by the FDA in November 2022 and by the EMA in February 2023.

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