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uniQure announces CMA from European Commission for HEMGENIX
The Fly

uniQure announces CMA from European Commission for HEMGENIX

uniQure N.V. announced that its partner, global biotechnology leader CSL, has received conditional marketing authorization, CMA, from the European Commission, EC, for HEMGENIX, the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B. HEMGENIX is approved for the treatment of adults with severe and moderately severe hemophilia B in adult patients without a history of Factor IX inhibitors. "The European approval of HEMGENIX represents another major milestone in the field of genomic medicine and innovation in the treatment of people living with hemophilia B," said Matt Kapusta, chief executive officer of uniQure. "This achievement is based on more than a decade of research and clinical development led by uniQure, and we are grateful for the tireless dedication of our employees, clinicians, patients and their families who made this possible." The European Commission’s decision follows the CHMP’s positive opinion in December 2022, based on findings from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date. The HOPE-B study 24-month analysis continued to show a sustained and durable effect of HEMGENIX. In a clinical setting, the treatment is generally well-tolerated with no serious treatment-related adverse events.

Published first on TheFly

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