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NICE Unconvinced on Cost-Effectiveness, Long-Term Benefits of CSL Behring's Hemgenix

NEW YORK – The National Institute for Health and Care Excellence on Wednesday said the National Health Service should not provide CSL Behring's one-time gene therapy Hemgenix (etranacogene dezaparvovec) to patients in England with moderately severe or severe hemophilia B. 

The lack of evidence on Hemgenix's long-term clinical effectiveness makes its cost-effectiveness estimates uncertain, NICE said in a draft guidance. With a list price of £2,600,000 ($3,304,730), the cost is also "above what NICE considers an acceptable use of NHS resources," NICE said.

Hemgenix, which the US Food and Drug Administration approved in 2022, is known as the most expensive drug in the world. The UK Medicines and Healthcare Products Regulatory Agency in March granted conditional marketing authorization to the gene therapy, and NICE stressed its draft guidance should not affect any Hemgenix treatment that NHS patients have already initiated.

Hemgenix is an adeno-associated virus vector-based gene therapy that delivers a functional copy of the factor IX (FIX) gene, which produces the eponymous blood-clotting protein missing or present at insufficient levels in hemophilia B patients.

To reach its decision, a NICE evaluation committee reviewed evidence from the ongoing Phase III, open-label HOPE-B trial, in which men with moderately severe or severe hemophilia B received Hemgenix after having FIX prophylaxis therapy for at least six months. Investigators compared patients' outcomes during the lead-in period on FIX prophylaxis therapy to their outcomes after receiving Hemgenix.

Seven to 24 months after treatment, patients experienced a 64 percent decrease in the adjusted annualized bleeding rate and a 73 percent decrease in the adjusted annualized bleeding rate for bleeds that needed FIX treatment. While the NICE committee agreed that the results suggest the drug reduced bleeding episodes during the follow-up period, experts raised doubts about whether patients would continue to experience benefits beyond 24 months. They concluded that the uncertainty related to outcomes durability could only be resolved by collecting long-term data.

The draft guidance is not NICE's final decision on the drug, and its recommendations may change after reviewing public comments. A second evaluation committee will meet Sept. 13, and a final decision from NICE is expected by Nov. 29.