European Commission Approves Pfizer's Hemophilia B Gene Therapy

Pfizer Inc. announced that the European Commission (EC) has granted conditional marketing authorization for DURVEQTIX® (fidanacogene elaparvovec). The gene therapy is indicated for adults with severe or moderately severe hemophilia B, a congenital deficiency in factor IX (FIX). DURVEQTIX aims to enable individuals to produce FIX themselves through a single-dose treatment, addressing the burden of frequent intravenous FIX infusions required by current standards of care.

The pivotal Phase 3 BENEGENE-2 study demonstrated significant reductions in bleeds post-treatment. After up to four years of follow-up, patients treated with DURVEQTIX experienced a median annualized bleed rate (ABR) of zero. This suggests a sustained protective effect against bleeds, potentially eliminating the need for ongoing prophylaxis treatments for eligible hemophilia B patients.

The therapy met its primary efficacy endpoint, showing non-inferiority and a statistically significant decrease in the ABR of total bleeds compared to standard prophylactic FIX replacement therapy. The treatment's efficacy remained stable from year two through year four post-administration. The safety profile was consistent with earlier study results and the therapy was generally well-tolerated.

This conditional marketing authorization is effective across all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. The approval follows earlier authorizations from the U.S. Food and Drug Administration (FDA) and Health Canada, where the therapy is marketed as BEQVEZTM. The EC's decision is based on the findings of the Phase 3 BENEGENE-2 study.