NICE rejects CSL Behring hemophilia B gene therapy

Aug. 4, 2023

U.K.’s price watchdog has recommended against reimbursement by England’s National Health Service (NHS) for CSL Behring and uniQure’s hemophilia B gene therapy, Hemgenix.

In draft guidance, a NICE committee said that while evidence from a clinical trial suggests that Hemgenix reduces the number of bleeding episodes a person has each year, there is not enough evidence related to long-term effectiveness. Because of those uncertainties and "some of the assumptions used to estimate cost effectiveness," the committee does not recommend the treatment.

Hemgenix got the nod for the U.S. FDA last November, and became the first gene therapy for adults with hemophilia B. Priced at $3.5 million per dose in U.S., it also became the most expensive drug in the world. 

In the NICE draft guidance, the committee noted that this cost is "above what NICE considers an acceptable use of NHS resources."

The historic U.S. approval was supported by results from the ongoing HOPE-B trial, which is the largest gene therapy trial in hemophilia B to date. Trial results showed that 94% (51 out of 54) of patients treated with Hemgenix were able to discontinue the use of prophylaxis and remained free of previous continuous routine prophylaxis therapy.  

Hemgenix was also approved by the European Commission for the EU and European Economic Area and was granted conditional marketing authorization by the UK's Medicines and Healthcare Product Regulatory Agency. 

NICE's opinion is not final though — the document was open for comments and a second committee evaluation meeting is schedules for September 13.