uniQure N.V. – a leading gene therapy company advancing transformative therapies for patients with severe medical needs – recently announced the achievement of a $100 million milestone associated with the first commercial sale of HEMGENIX in the United States by its partner CSL. HEMGENIX (etranacogene dezaparvovec) is known as a one-time administered gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.
uniQure had conducted the multi-year research and clinical development program for HEMGENIX, which included 3 clinical trials across 34 global sites and involved 67 adults with hemophilia B. And in May 2021, uniQure and CSL completed a licensing transaction providing CSL Behring with exclusive rights to commercialize and continue clinical development of HEMGENIX globally. Plus uniQure is responsible for the global manufacturing of the product at its licensed facility in Lexington, Massachusetts. Under the agreement with CSL Behring, the milestone payment is due within 30 days after the achievement of the milestone.
HEMGENIX is the first approved gene therapy for hemophilia B in the United States, European Union (EU) and European Economic Area (EEA), and the UK.
KEY QUOTES:
“The first commercial sale in the U.S. is a major milestone for uniQure as it marks the fulfillment of our promise to deliver genetic medicines that have the potential to transform people’s lives. uniQure’s successful development of HEMGENIX further validates our AAV platform built on the back of 25 years of scientific leadership and innovation in the field of gene therapy. We look forward to our continued collaboration with CSL Behring as they work to bring this important treatment to those living with hemophilia B.”
– Matt Kapusta, chief executive officer of uniQure
