London, UK – The National Institute for Health and Care Excellence (NICE) has rejected CSL Behring’s gene therapy, Hemgenix, as a treatment option for severe and moderately severe haemophilia B.
Despite promising results from the phase 3 HOPE-B trial, where 96% of patients no longer required routine FIX prophylaxis after a single infusion, NICE’s draft decision disappoints the company. Hemgenix had received conditional marketing authorization from the Medicines and Healthcare products Regulatory Agency in early 2023, making it the first gene therapy licensed for haemophilia B in the UK.
Approximately 2,000 UK patients suffer from this rare bleeding disorder, and the rejection leaves them seeking other treatment options.
The drug gained Worldwide Notice after it was approved by the FDA in November 2022 and has already been administered to patients in the U.S. The drug will sports an eye-popping $3.5 million per dose price tag, making it the worlds most expensive drug.
