The National Institute for Health and Care Excellence (NICE) have released the final appraisal documentfor the recommendation of gilteritinib, a highly selective, second generation FLT3 inhibitor for adults with relapsed/refractory FLT3-mutated acute myeloid leukemia (AML), making it the first oral monotherapy to be approved in the United Kingdom for this patient population. However, the recommendation does not extend to maintenance use after hematopoietic stem cell transplant. 1
This recommendation is based on results from the phase III ADMIRAL trial ( NCT02421939), which demonstrated a significantly longer median overall survival (9.3 vs5.6 months; p < 0.001) and deeper responses for patients treated with gilteritinib compared with salvage chemotherapy, respectively. One-year overall survival was also higher for patients treated with gilteritinib compared with salvage chemotherapy (37.1% vs16.7%, respectively; HR 0.64; 95% CI, 0.49–0.83). For more information on the results from this trial, read our summary article here.
Gilteritinib has already received approval as a single agent from the U.S. Food and Drug Administration ( FDA) and the European Medicines Agency ( EMA), based on results from the same trial.