FDA Accepts Hemophilia B Gene Therapy Product for Priority Review

uniQure N.V., in partnership with CSL Behring, has announced that etranacogene dezaparvovec, its gene therapy product for hemophilia B, has been accepted for priority review by the U.S. Food and Drug Administration (FDA). If approved, the drug would be the first available gene therapy treatment for hemophilia B. The acceptance for review is based on substantial data including the promising results generated from uniQure’s HOPE-B pivotal study, the largest gene therapy trial in hemophilia B to date.

Etranacogene dezaparvovec has been shown in clinical trials to significantly reduce the rate of annual bleeds in trial participants after a single one-time infusion. A priority review  by the FDA is reserved for medicines that, if approved, would be potentially 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 in 6 months as compared to 10 months for standard review.

Gene therapy, as described in uniQure’s announcement of the acceptance for review, has the potential to enable more normal clotting in patients with hemophilia B. Gene therapy achieves this with modified non-infectious viruses that serve as “vectors” that can enter certain cells. Vectors carry genetic instructions to specific cells. Once delivered, the new genetic instructions allow the cellular machinery to produce their own stable levels of FIX. A certain type of vector, called an adeno-associated virus, or AAV, dissolves after delivering its genetic instructions. These genetic instructions remain in the target cells, but never actually become a part of a person’s own DNA.

You can learn more about this important development by reading uniQure’s full announcement at https://www.globenewswire.com/news-release/2022/05/24/2449266/0/en/uniQure-Announces-FDA-Acceptance-of-Biologics-License-Application-for-Etranacogene-Dezaparvovec-under-Priority-Review.html.

The Coalition for Hemophilia B joins the entire community in heralding this potentially major advance in treatment. We will continue to provide our members with updates on the review process and other treatment advances as information becomes available. Please follow our social media in addition to our print and electronic publications for the latest news.