ABPI cheers new gene therapy for IMF and innovative payment first

NICE has recommended CSL Behring’s HEMGENIX® (etranacogene dezaparvovec) as the first single infusion gene therapy for adults with severe or moderately severe haemophilia B without a history of FIX inhibitors for reimbursement via a managed access agreement, recognising the treatment as cost-effective in England [1].

Responding to the news, Paul Catchpole, Director of Value and Access policy, said:

“This announcement is positive news for eligible patients with haemophilia B, as well as the wider gene therapy community, which is striving to bring these highly innovative treatments to patients.

“It marks the first time a medicine has been granted managed access through the Innovative Medicines Fund since it was launched in 2022. We hope we will see more new medicines reaching patients through this pathway in the future”.

“It is also encouraging to see an innovative payment model has been agreed, with payments potentially linked to how well the treatment performs over time. Looking at the new medicines pipeline, these payment models may become more important for some medicines in the years ahead.”