UniQure Needs Longer-Term Data For Hemophilia B Gene Therapy EtranaDez

The delay in uniQure N.V.’s filing plans for its hemophilia B gene therapy etranacogene dezaparvovec reflects changing requests from the US Food and Drug Administration, which could affect players across the hemophilia A and B space – but does not reflect any negatives for uniQure’s product itself. The latest Phase III data show sustained increases in Factor IX (FIX) activity a year after administration of the therapy.

At its R&D meeting on 22 June, the Amsterdam-headquartered company announced an update from the Phase III HOPE-B trial and said that after it and partner CSL Limited (CSL Behring) communicated with the FDA, it would use a non-inferiority analysis of annualized bleeding rates (ABR) as