Etranacogene Dezaparvovec Shows Sustained Efficacy and Safety in Adult Patients with Severe or Moderately Severe Haemophilia B 3 Years after Administration in the HOPE-B Trial

Introduction Etranacogene dezaparvovec (formerly AMT-061) is the first approved gene therapy for haemophilia B in the EU and US. The HOPE-B pivotal phase 3 clinical trial (NCT03569891) demonstrated superior bleed protection compared to FIX prophylaxis up to 24 months post treatment with ongoing follow-up from Year 2 onward. Here, we report efficacy and safety during Years 1-3.