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UK's NICE Backs Astellas' Xospata for FLT3-Mutated AML in Proposed Guidance

NEW YORK – The National Institute for Health and Care Excellence (NICE) in the UK on Thursday published a final draft guidance recommending that the National Health Service make available gilteritinib (Astellas' Xospata) monotherapy for patients with FLT3-mutated acute myeloid leukemia.

In England, around 16 percent of female and 15 percent of male AML patients survive for more than five years, and patients who harbor a FLT3 mutation are more likely to relapse. There are limited treatment options for relapsed or refractory AML: salvage chemotherapy can be used to manage the disease, for example, but it can also cause unpleasant side effects that can send patients to the hospital.

Gilteritinib is the first oral monotherapy to be approved in the UK that targets relapsed or refractory FLT3-mutated AML and can be taken by patients at home rather than in the hospital.

The drug was approved by the European Medicines Agency last year for relapsed or refractory FLT3-mutated AML based on results of the randomized, Phase III ADMIRAL study, which showed an average overall survival of 9.3 months for those treated with gilteritinib compared to 5.6 months for patients on salvage chemotherapy.

The list price for gilteritinib is £14,188 ($17,860) per 28-day pack, but NICE said that Astellas made gilteritinib available to the NHS at a discount.

In January, NICE determined that the drug was not cost effective, which prompted Astellas to provide additional data and revise its cost-effectiveness estimates. "Gilteritinib is now considered a cost-effective use of NHS resources," NICE said in a statement, estimating that more than 400 AML patients may be eligible to receive the treatment each year through the NHS once the final guidance has been issued.

However, NICE noted that there is still uncertainty around long-term survival of patients post stem cell transplant. As such, it is not recommending that gilteritinib be administered as maintenance therapy in this setting.

NICE's final appraisal document will now be sent to consultees and commentators who have until July 30 to appeal or correct errors. The agency expects to issue a final guidance on August 12.