Gene therapy involves adults with haemophilia B and brings renewed hope to patients across Europe

CSL has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of etranacogene dezaparvovec.

It concerns the conditional marketing authorisation (CMA) of the therapy, which reduces the rate of annual bleeds with a single infusion, among adults with haemophilia B. If the marketing authorisation is successfully approved by the European Commission (EC) – under the brand name HEMGENIX – it would become the first gene therapy for people living with haemophilia B in the EU and European Economic Area.

A positive CHMP opinion followed findings from the HOPE-B trial, the largest gene therapy trial in haemophilia B to date. These findings demonstrated that haemophilia B patients, when treated with etranacogene dezaparvovec, showed stable increases in mean factor IX activity levels, which then led to an adjusted annualised bleed rate reduction of 64%. Meanwhile, following the infusion of etranacogene dezaparvovec, 96% of patients discontinued routine FIX prophylaxis.

The 24-month HOPE-B analysis also showed that in a clinical setting etranacogene dezaparvovec continued to be generally well-tolerated with no serious adverse events.

Emmanuelle Lecomte Brisset, senior vice president at CSL, explained: “The CHMP’s positive opinion moves us one step closer to bringing this ground-breaking innovation to haemophilia B patients in Europe.”

“Getting a new medicine to this stage of the regulatory process takes the support of many, including clinical trial participants, the haemophilia community in general, investigators, clinicians, regulatory agencies, our people, and our partners at uniQure to name a few,” he added.

The positive opinion from the CHMP will now be reviewed by the EC, which has the authority to approve medicines for EU member states.

Haemophilia B is a rare, life-threatening disease and people with the condition are particularly vulnerable to bleeds in their joints, muscles and internal organs, leading to pain, swelling and joint damage.