Business
uniQure Announces FDA Acceptance of Biologics License Application for Etranacogene Dezaparvovec under Priority Review
If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with hemophilia B LEXINGTON, Mass. and AMSTERDAM, May 24, 2022
About this update from Uniqure N.v.
[{"type":"text","content":"If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with hemophilia B\nLEXINGTON, Mass. and AMSTERDAM, May 24, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) submitted by CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for the treatment of adults with hemophilia B. CSL Behring is uniQure’s global commercialization partner for etranacogene dezaparvovec and is solely responsible for all regulatory activities, including filings and agency interactions, associated with etranacogene dezaparvovec. uniQure led the multi-year clinical development of etranacogene dezaparvovec, including the pivotal Phase III HOPE-B clinical trial, and the company will be responsible for the global commercial supply of etranacogene dezaparvovec. \"Etranacogene dezaparvovec has the potential to be the first gene therapy approved in hemophilia B and the acceptance of the BLA marks an important milestone in uniQure’s mission of delivering the promise of gene therapy to people living with rare diseases,” stated Matt Kapusta, chief executive officer of uniQure. “uniQure has been a leader in gene therapy for nearly 25 years and, if approved, etranacogene dezaparvovec would represent our second gene therapy to complete its journey to patients. We are proud of this accomplishment, which is a culmination of significant contributions from all our employees, clinical investigators and the hemophilia community. We are pleased with the promising results generated from our HOPE-B pivotal study, the largest gene therapy trial in hemophilia B to date and, assuming FDA approval, we look forward to partnering with our colleagues at CSL Behring to bring this life-changing treatment option to people 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 trial par...