FDA accepts BLA for etranacogene dezaparvovec for priority review for treatment of Hemophilia B.- CSL Behring.
CSL Behring, a business of CSL, announced that the FDA has accepted its Biologics License Application (BLA), for priority review, for etranacogene dezaparvovec (also known as CSL 222), an investigational gene therapy for the treatment of adults with hemophilia B.
Etranacogene dezaparvovec was specifically designed to make near-normal blood-clotting ability possible by addressing the underlying cause of hemophilia B: a faulty gene that causes a deficiency in clotting Factor IX (FIX). Etranacogene dezaparvovec has been shown in clinical trials to significantly reduce the rate of annual bleeds in people with hemophilia B after a single one-time infusion, and if approved, would be the first ever gene therapy treatment option for the hemophilia B community.
The BLA is supported by results from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date. People with Hemophilia B treated with etranacogene dezaparvovec demonstrated reduced adjusted annualized bleeding rate (ABR) by 64% and superiority to prophylaxis treatment at 18 months post-treatment compared to a 6-month run in period (p=0.0002). The multi-year clinical development program for etranacogene dezaparvovec was led by uniQure and sponsorship of the clinical trials has transitioned to CSL Behring after its acquiring global rights to commercialize the investigational treatment.
Priority review of a BLA is reserved for medicines that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. Upon acceptance for priority review, the FDA goal is to take action on the BLA in 6 months as compared to 10 months for standard review. Previously, the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for etranacogene dezaparvovec under its accelerated assessment procedure.
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