Core Insights - CSL announced the four-year results from the pivotal HOPE-B study, confirming the long-term durability and safety of HEMGENIX for adults with hemophilia B [1][4] - HEMGENIX is the first gene therapy approved for hemophilia B, providing significant long-term bleed protection and reducing the need for routine factor IX prophylaxis [1][11] Study Results - The HOPE-B trial involved 54 adult male participants, with 51 completing four years of follow-up [2][8] - Mean factor IX levels were sustained at 37.4 IU/dL at year four, with a 90% reduction in the adjusted annualized bleeding rate (ABR) from 4.16 (lead-in) to 0.40 [2][5] - Joint bleeds decreased from a mean ABR of 2.34 at lead-in to 0.09 during year four, with 94% of patients remaining free of continuous prophylaxis [2][5] Safety Profile - No serious adverse events related to HEMGENIX were reported, and it was generally well-tolerated [3][9] - The most common treatment-related adverse events were increases in alanine transaminase (ALT), with 16.7% of participants requiring supportive care [3][16] Company Commitment - CSL is dedicated to ongoing data analyses of HEMGENIX to provide healthcare providers and patients with necessary information for informed treatment decisions [4][20] - The company has established a post-marketing registry to gather additional evidence on the long-term safety and efficacy of HEMGENIX [4][20]
CSL Behring's Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Four Years Post-Infusion Data Continue to Show Sustained Efficacy and Safety in Adults with Hemophilia B