KING OF PRUSSIA, PA — Global biotechnology leader CSL (ASX: CSL) and uniQure N.V. (NASDAQ: QURE) recently announced that the companies have received the 2023 Prix Galien USA Award in the category of Best Product for Rare/Orphan Diseases for HEMGENIX® (etranacogene dezaparvovec-drlb).
CSL received approval from the U.S. Food and Drug Administration (FDA) for HEMGENIX in November 2022. It is the first and only gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes.
“CSL is humbled and honored to receive a Prix Galien award for HEMGENIX,” said Dr. Bill Mezzanotte, Head of Research and Development, CSL. “We thank The Galien Foundation for acknowledging the enormous promise and hope that HEMGENIX offers to eligible people living with hemophilia B.”
The multi-year clinical development of HEMGENIX was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment.
“HEMGENIX represents a major milestone in the field of genomic medicine and ushers in a new treatment paradigm for eligible people living with hemophilia B,” said Matt Kapusta, Chief Executive Officer, uniQure. “It gives us great pride that after many years of development, this important gene therapy is now being made available by CSL to patients in geographies around the world.”
HEMGENIX also has been granted conditional marketing authorization by the European Commission (EC) for the European Union and European Economic Area, and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA), as well as authorization by Health Canada.
The Prix Galien USA is America’s preeminent prize acknowledging the leading-edge of scientific advances in life sciences.
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