uniQure recently announced publication of the clinical trial results associated with approval of the hemophilia gene therapy product Hemgenix®. The data, which were published in the New England Journal of Medicine, (NEJM), were drawn from the HOPE-B Trial program, which has been investigating the therapy in patients with severe and moderately severe hemophilia B.

Hemgenix was approved by the U.S. Food and Drug Administration in November 2022. Clinical trial development of the therapy was led by uniQure, while global rights to commercialize the product are held by CSL Behring. It is currently indicated for the treatment of adults with hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

According to a new uniQure press release, the HOPE-B trial results published in the NEJM show the annualized bleeding rate of spontaneous bleeding episodes and all joint bleeding episodes decreased by 71% and 78% respectively, from lead-in period to post-treatment. In addition, 96.3% of patients discontinued prophylaxis with factor IX replacement therapy from day 21 through month 18 post-treatment. Annualized factor IX infusions also significantly decreased from 72.5 infusions per participant during lead-in period to 2.5 infusions post-treatment.    

“The results published in NEJM add to the established body of evidence demonstrating the long-term efficacy and safety of HEMGENIX® and confirm that this innovative new medicine not only restores blood clotting factor to near normal levels and significantly reduces factor use, but also that gene therapy may reduce the burden of care and improve quality of life for people living with this life-long condition,” said Dr. Steven Pipe, Professor and the Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology at the University of Michigan and lead investigator of the HOPE-B study. “HOPE-B was also the first and only phase 3 study to demonstrate efficacy of a gene therapy for hemophilia B in individuals with circulating neutralizing antibodies that have the potential to interfere with the effects of treatment. Results from HOPE-B suggest that HEMGENIX® may be effective in a broad range of hemophilia B patients, regardless of prior exposure to common adeno-associated viruses.”

Source: uniQure press release dated February 23, 2023