The Food and Drug Administration has approved the addition of overall survival data in labelling for gilteritinib, indicated for adult patients who have relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

Approval was based on the ADMIRAL trial (NCT02421939), which included 371 adult patients with relapsed or refractory AML having a FLT3 ITD, D835, or I836 mutation by the LeukoStrat CDx FLT3 Mutation Assay.

Patients were randomised (2:1) to receive gilteritinib 120 mg once daily (n=247) over continuous 28-day cycles or pre-specified salvage chemotherapy (n=124).

Salvage chemotherapy included either intensive cytotoxic chemotherapy or a low-intensity regimen.

For the analysis, overall survival (OS) was measured from the randomisation date until death by any cause.

The median OS was 9.3 months for patients receiving gilteritinib and 5.6 months for those on the chemotherapy arm (HR 0.64; 95% CI: 0.49,0.83; 1 sided p-value=0.0004).

The results were consistent in the intensive chemotherapy stratum (HR 0.66; 95% CI: 0.47-0.93) and the low-intensity regimen stratum (HR 0.56; 95% CI: 0.38-0.84).

The adverse reactions occurring in at least 20% of patients receiving gilteritinib were increased transaminase, myalgia/arthralgia, fatigue/malaise, fever, mucositis, edema, rash, noninfectious diarrhoea, dyspnea, nausea, cough, constipation, eye disorders, headache, dizziness, hypotension, vomiting, and renal impairment.

Prescribing information contains a Boxed Warning alerting health care professionals and patients about the risk of differentiation syndrome, which may be life-threatening or fatal if not treated.

The recommended gilteritinib dose is 120 mg orally once daily.

Source: FDA