NICE recommends Astellas’ Xospata for NHS use

The UK’s National Institute for Health and Care Excellence (NICE) has recommended Astellas’ Xospata for routine use on the NHS, backtracking on its previous rejection of the drug in January.

At the beginning of the year, NICE rejected Xospata (gilteritinib) citing uncertainty around the long-term survival benefit of the drug in FLT3-positive acute myeloid leukaemia (AML) patients as grounds for the negative decision. The cost-effectiveness watchdog also said that the FLT3-inhibitor was too expensive, at a list price of £14,188 per 28-day pack.

However, following further discussions with Astellas, NICE has recommended Xospata monotherapy for treating adult patients with relapsed or refractory FLT3-positive AML. Although those discussion are confidential, Astellas has agreed a commercial arrangement with the NHS to make Xospata available to the NHS with a discount.

NICE also highlighted that while FLT3-positive AML is usually treated with salvage chemotherapy, Xospata is an important alternative treatment, given that it can be taken as an oral tablet at home. It also acknowledged that the clinical evidence demonstrates that those taking Xospata live longer compared with people having salvage chemotherapy.

Still, NICE maintained that there is ‘considerable uncertainty’ about the long-term survival benefit of Xospata, and said there is no robust evidence of further benefit if a patient restarts the drug after a stem cell transplant.

Despite the concerns, the drug ultimately meets NICE’s life-extending criteria for end-of-life treatment, and cost-effectiveness estimates are now in range with what NICE usually considers an acceptable use of NHS resources.

Between 20-30% of people with AML have FLT3 mutations, which are often associated with poorer outcomes, including a higher relapse rate, faster progression and shorter overall survival times.

In the phase 3 ADMIRAL study, Xospata extended overall survival to 9.3 months compared to 5.6 months in the salvage chemotherapy treatment group. After one year of treatment, 37% of patients taking Astellas’ drug were still alive compared to 17% of those in the chemotherapy arm.

“This marks a pivotal moment as, for the first time, adults in the UK with this specific form of AML have a much needed option at the point when current treatment no longer works or they relapse,” said Jackie Williams, general manager for Astellas in the UK. “We are proud to bring such an important treatment option that could significantly extend the lives of these patients.”