FDA accepts Pfizer’s hemophilia B (PFE) gene therapy application.

Pfizer PFE announced that its applications for approval in the United States and the European Union (EU) for the treatment of hemophilia B gene therapy fidanacogene elaparvovec have been accepted for review.

While a final decision from the FDA is expected in the second half of 2024, a decision from the European Medicines Agency (EMA) is expected in mid-2024.

Regulatory filings for fidanacogene elaparvovec in both markets are based on data from the Phase III BENGENE-2 study. The study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post infusion with fidanacogene elaparvovec compared to study participants administered the current standard of care (SOC) treatment i.e., a regimen of prophylaxis with Factor IX (FIX).

Year-to-date, Pfizer shares have lost 28.9% versus the industry’s 0.7% growth.

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Fidanacogene elaparvovec is a single-use gene therapy product consisting of a bioengineered AAV capsid and a highly active human coagulation FIX gene. The treatment aims to allow patients to produce FIX on their own without the need to receive exogenous FIX.

Pfizer licenses the elaparvovec fidanacogene gene therapy from Spark Therapeutics, a subsidiary of Roche RHHBY. The agreement was entered into in 2014, five years before Roche’s acquisition of Spark Therapeutics in 2019. Under the terms of the licensing agreement with the Roche subsidiary, Pfizer is solely responsible for all pivotal studies and the potential commercialization of fidanacogene elaparvovec.

If approved, Pfizer’s gene therapy will face competition from Hemgenix, a one-time gene therapy that was approved by the FDA last November for the treatment of hemophilia B in patients over the age of 18. Hemgenix was developed by uniQure QURE in partnership with CSL Behring, the latter a subsidiary of CSL Limited. Following approval, uniQure/CSL’s Hemgenix became the first FDA-approved gene therapy for the treatment of hemophilia B. In February, the treatment also received approval in the EU.

The FDA approval of uniQure/CSL Hemgenix was based on results from the pivotal HOPE-B study, which showed that following gene therapy infusion, patients produced an average FIX activity of 39% at six months and 36.7% at 24 months. The mean adjusted ABR for all bleeds was also reduced by 54% after 7-18 months of Hemgenix administration. The clinical development program for Hemgenix was initially led by uniQure, which was later transferred to CSL after it acquired the global rights to commercialize the treatment.

In addition to fidanacogene elaparvovec, Pfizer is developing another gyroctocogene fitelparvovec gene therapy for the treatment of hemophilia A. The therapy was developed in collaboration with Sangamo Therapeutics GMOs. Sangamo and Pfizer are evaluating the gyroctocogene fitelparvovec in the Phase III AFFINE study. Pfizer/Sangamo expects to report pivotal data from the AFFINE trial by mid-2024. Based on these data, Sangamo/Pfizer expects to begin regulatory filing for gene therapy candidate in the EU and US during the second half of 2024.

In addition to gene therapies, Pfizer is also evaluating marstacimab, an investigational inhibitor of the anti-tissue factor pathway, to treat patients with hemophilia A and B with and without inhibitors in the ongoing Phase III BASIS study. Last month, Pfizer announced that the BASIS trial met its primary endpoints. The study data showed that marstacimab treatment achieved a statistically significant and clinically relevant reduction in ABR compared with current SOC treatment for patients with haemophilia, during a 12-month period.

A rare genetic blood disorder, hemophilia is caused by a missing clotting protein (FVIII in case of hemophilia A and FIX in case of hemophilia B), which prevents normal blood clotting. If the blood doesn’t clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage.

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