Pfizer Inc. (NYSE: NYSE:PFE) has received conditional marketing authorization from the European Commission for its gene therapy product DURVEQTIX, designed for the treatment of severe and moderately severe hemophilia B in adult patients who do not have a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74.
The authorization is based on the results from the Phase 3 BENEGENE-2 study, which showed that a one-time dose of DURVEQTIX significantly reduced the annualized bleed rate (ABR) compared to the current standard of prophylactic infusions of factor IX (FIX). The study met its primary efficacy endpoint of non-inferiority and demonstrated a statistically significant decrease in ABR for total bleeds post-treatment.
Patients treated with DURVEQTIX saw a median ABR of zero with a range of 0 to 9.9 bleeds after up to four years of follow-up, indicating sustained bleed protection and the potential to reduce the treatment burden associated with regular intravenous FIX infusions.
DURVEQTIX was generally well-tolerated, with a safety profile consistent with earlier phase results. The most common adverse reaction was an increase in liver enzymes, managed with corticosteroids. No serious adverse events related to the treatment or associated with infusion reactions, thrombotic events, or FIX inhibitors were reported.
The approval extends to all 27 European Union member states, as well as Iceland, Liechtenstein, and Norway, following similar approvals by the U.S. Food and Drug Administration (FDA) and Health Canada.
The European Marketing Authorization Application included data from the BENEGENE-2 study when 41 participants had reached 15 months of follow-up, with a subsequent data cut monitoring some patients for up to four years. Clinical trial participants will continue to be observed for a total of 15 years to assess the long-term safety and efficacy of the therapy.
Pfizer has a history of over 40 years in hemophilia treatment advancements and continues to expand its portfolio, including recent positive results from a Phase 3 program for hemophilia A gene therapy and a Phase 3 trial for marstacimab, an investigational treatment for hemophilia A and B.
InvestingPro Insights
As Pfizer Inc. (NYSE:PFE) secures a new milestone with the European Commission's conditional marketing authorization for its hemophilia B gene therapy, DURVEQTIX, investors may find the following insights from InvestingPro valuable for their assessment of the company's financial health and market performance.
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