NEW YORK – Patients in England will be able to access CSL Behring's hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) under a first-of-its-kind managed access agreement the company has reached with the UK National Health Service.
Managed access is a temporary agreement under which patients can receive promising treatments that otherwise might not be recommended due to uncertain clinical or cost-effectiveness evidence. During the agreement period, drugmakers collect additional data, which the UK National Institute for Health and Care Excellence uses to evaluate if the treatment should be made routinely available at NHS England.
Hemgenix is a one-time gene therapy that aims to treat moderately severe to severe hemophilia B by delivering a functional copy of the factor IX (FIX) gene that produces a protein needed for blood clotting. As of last year, the institute was unconvinced that the cost and long-term benefits of Hemgenix, which has a list price of £2.6 million ($3.3 million), were an appropriate use of NHS resources.
However, last week NICE issued a final draft guidance announcing that the institute and NHS England had reached a managed access agreement with CSL Behring, under which it could now recommend Hemgenix be available to patients at eight specialist centers in England while more data are collected. Under the managed access agreement, Hemgenix, which has conditional marketing authorization from the UK Medicines and Healthcare Products Regulatory Agency, will continue to be available to patients through the Innovative Medicines Fund.
CSL Behring will collect data on the gene therapy's long-term treatment durability and the proportion of patients who continue to require the current standard of care — factor IX prophylaxis — after receiving Hemgenix through the ongoing Phase III HOPE-B clinical trial and within an open-label extension study. The firm will also collect data on Hemgenix's activity from clinical practices in the UK.
The agreement stipulates that CSL Behring will submit additional clinical trial results and an updated economic model to NICE by November 2027. The Phase III clinical trial will wrap up in 2030. Hemgenix is the first treatment to enter NHS England's Innovative Medicines Fund under a managed access scheme. NHS England launched the Innovative Medicines Fund in 2022 to provide patients access to therapeutic advances outside of the cancer setting, including cell and gene therapies, at a "responsible price."
"This transformative gene therapy is the first of its kind for hemophilia B patients on the NHS," Stephen Powis, national medical director at the NHS, said in a statement. The gene therapy "has the potential to significantly improve the lives of hundreds of people by helping to reduce symptoms such as painful bleeds."
Hemgenix has also received regulatory approval in the US and Canada.