CSL Behring, a business of CSL, announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA), for priority review, for etranacogene dezaparvovec (also known as CSL222), 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.
"FDA's acceptance of the etranacogene dezaparvovec BLA for priority review is another pivotal milestone in CSL Behring's commitment to deliver innovative treatment options that address unmet needs for people living with rare diseases," said Brahm Goldstein, M.D., Vice President, Research and Development, Hematology at CSL. "For more than 35 years, CSL Behring has been at the forefront of innovation in the treatment of hereditary bleeding disorders, offering the largest portfolio of treatment options for the community including a novel, long-acting prophylaxis FIX replacement treatment. We look forward to continuing to work with the FDA throughout the review process of the BLA and with our partners at uniQure to potentially provide a new transformative treatment option to people with hemophilia B."
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 (Nasdaq: QURE) 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.
"The acceptance of etranacogene dezaparvovec for review by the FDA brings us closer to our goal of delivering a life-changing treatment option for people with hemophilia B," said Bill Mezzanotte, M.D., MPH, Executive Vice President, Head of R&D and Chief Medical Officer for CSL. "Etranacogene dezaparvovec, potentially the first gene therapy approved for hemophilia B, further demonstrates CSL's promise to relentlessly pursue innovative and disruptive technologies when it benefits rare and serious disease patients with unmet medical needs."
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