Xospata(gilteritinib)

Xospata (gilteritinib) is indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation, as detected by an FDA-approved test. The drug received initial US approval in 2018, with an approval date of May 29, 2019, under Application Number NDA 211349 S-001. The recommended starting dose is 120 mg orally once daily.

In a study with a median follow-up of 17.8 months, gilteritinib demonstrated an Overall Survival Hazard Ratio of 0.64 (95% CI: 0.49 – 0.83) with a p-value of 0.0004. The FDA-adjudicated complete remission (CR) rate in the gilteritinib arm was 14.2%, compared to 10.5% in the chemotherapy arm. The CR/CRh rate in the gilteritinib arm was 22.6%, with a median duration of response of 7.4 months and a median time to first response of 2 months.

Notable safety findings include a 3% incidence of Differentiation Syndrome, with an 82% recovery rate. Posterior Reversible Encephalopathy Syndrome (PRES) incidence was 1%, and QTc interval >500 msec incidence was 1%. Pancreatitis occurred in 4% of patients. Fatal adverse reactions were observed in 2% of patients, while dose interruption, dose reduction, and treatment discontinuation due to adverse reactions occurred in 29%, 6%, and 7% of patients, respectively.