Treatment is the only single infusion gene therapy for the treatment of severe haemophilia B
CSL has announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorisation to Hemgenix.
Also known as etranacogene dezaparvovec, the therapy is the only single infusion gene therapy for the treatment of severe and moderately severe haemophilia B. It involves adults without a history of Factor IX (FIX) inhibitors across England, Scotland and Wales.
The decision follows conditional marketing authorisation from the European Commission – which also includes Northern Ireland and the Republic of Ireland – and is based on data from the ongoing phase 3 single-dose, multi-centre HOPE-B trial. The study has incorporated 54 participants – the largest gene therapy trial in haemophilia B to date.
Results from the trial demonstrate that haemophilia B patients treated with a single infusion of etranacogene dezaparvovec showed considerable increases in mean FIX activity levels of 36.9% at 18 months. In addition, these were sustained at 36.7% at 24 months post-treatment, compared to the six-month lead-in period.
The multi-year clinical development of etranacogene dezaparvovec was led by uniQure and sponsorship of the clinical trials transitioned to CSL after the company received licenced global rights to commercialise the treatment.
Stephen Allan, country manager, of CSL Behring UK and Ireland, reflected: “Today’s decision represents an important milestone for a potential new therapeutic option for people living with haemophilia B in the UK. We are now committed to working collaboratively with the National Institute for Health and Care Excellence to provide access to this innovative treatment for eligible patients across the UK.”
Dr Bill Mezzanotte, head of research and development and chief medical officer at CSL, explained: “The positive decision from the MHRA is the essence of great science delivering a medicine that we believe could potentially change the treatment paradigm for both people living with haemophilia B and the healthcare professionals who treat them.”
He added: “This asset, and our partnership with uniQure, underscore CSL’s promise to pursue, develop and deliver new treatments, particularly in disease states we know well like haemophilia B.”
Haemophilia is characterised by bleeding episodes and long-term complications such as joint damage. Nearly 2,000 people live with haemophilia B in the UK and those with the severe disease currently require lifelong treatment.
