The U.K.’s cost-effectiveness watchdog has tossed cold water on CSL Behring and uniQure’s pricey new hemophilia B gene therapy Hemgenix.
In draft guidance, the National Institute for Health and Care Excellence (NICE) recommended against Hemgenix reimbursement by England’s National Health Service (NHS) after finding uncertainty about the drug's long-term effectiveness.
Specifically, NICE took issue with a comparison between the drug and the standard of care factor IX prophylaxis. The comparison points to improved bleeding outcomes for Hemgenix, but it leaves some doubts due to differences in study methods, NICE said.
The cost-effectiveness estimates for the new therapy are also murky, NICE added.
In the U.K., the therapy comes with a 2.6-million-pound ($3.3 million) price tag. However, CSL reached a “commercial arrangement” that would take effect if Hemgenix, or etranacogene dezaparvovec, were to be recommended.
For its part, CSL is “disappointed” with the decision but “remains confident” in the med’s benefits and long-term value, a company spokesperson said in an emailed statement.
"We firmly believe in the potential of etranacogene dezaparvovec to bring a change in the lives of eligible haemophilia B patients, offering them possible long-lasting therapeutic benefit,” CSL’s U.K. and Ireland general manager, Eduardo Cabas, added in the statement. "NICE has expressed its commitment to a more progressive approach when assessing highly innovative medicines, and CSL Behring asks NICE to honour this commitment when assessing etranacogene dezaparvovec and other gene therapies.
Nothing is set in stone yet. The draft guidance is open to comment for the next 15 days before the NICE committee meets to reassess its initial recommendation based on the additional input. A final decision will be made in the fall.
Hemgenix won conditional marketing authorization from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) in March after scoring the same from the European Commission in February. It’s the world's only one-time gene therapy for the bleeding condition and is meant to replace the standard, lifelong treatment of factor IX prophylaxis.