Phase III data on Hemgenix published in NEJM

Today, Dutch gene therapy company uniQure (Nasdaq: QURE) announced that the prestigious New England Journal of Medicine (NEJM) has published data from the pivotal Phase III HOPE-B study evaluating the efficacy, durability and safety of Hemgenix (etranacogene dezaparvovec-drlb), the first gene therapy approved for the treatment of adults with hemophilia B.

Earlier this week, the  European Commission approved Hemgenix, dubbed the world’s costly drug, and out-licensed globally to Australian biotech CSL Limited (ASX: CSL), which follows the US approval in November 2022. The approval applications were supported by the HOPE-B study.

Results from the HOPE-B trial, the largest gene therapy study in hemophilia B to-date, showed that Hemgenix is superior to routine factor IX (FIX) prophylaxis in Annualized Bleeding Rate (ABR), FIX activity, FIX therapy consumption, FIX infusion rate, and spontaneous and joint bleeding ABR.