Gilteritinib (Xospata)

Key Messages

• Canada’s Drug Agency (CDA-AMC) recommends that Xospata be reimbursed by public drug plans as posttransplant maintenance for measurable residual disease (MRD)–positive, FLT3 internal tandem duplication (ITD)–mutated acute myeloid leukemia (AML), if certain conditions are met.
• The pan-Canadian Oncology Drug Review Expert Review Committee (pERC) determined that Xospata demonstrates acceptable clinical value compared with active surveillance in patients with MRD-positive, FLT3 ITD–mutated AML. A substantial unmet clinical need in the MRD-positive, post–allogeneic hematopoietic cell transplantation (allo-HCT) setting was identified by pERC due to the severity of the disease, specifically the poor outcomes associated with relapsed or refractory AML, as well as the lack of approved or funded treatment options. This determination was sufficient for pERC to recommend that Xospata be reimbursed. Given that Xospata is expected to be an additive treatment to active surveillance, acceptable clinical value refers to added value versus active surveillance.
• Evidence from a post hoc subgroup analysis of a clinical trial suggested that 24 months of treatment with Xospata may improve the length of time patients stay in remission and live longer. The committee considered the results from this study to be highly uncertain, hypothesis-generating, and inconclusive based on the statistical analyses; however, pERC also noted that the results are clinically important for this population due to the biologic plausibility that Xospata would be effective in this population, given that MRD positivity is a known prognostic factor for relapse in patients with FLT3 ITD–mutated AML.
• Patients and clinicians identified a need for effective and well-tolerated therapies that can be safety administered following transplant to eradicate residual disease, as well as treatments that result in improved health-related quality of life (HRQoL). Xospata may meet some of the needs identified, providing a new therapy for patients who are MRD-positive in the post–allo-HCT maintenance setting that may delay or prevent relapse. No definitive conclusion could be reached regarding the effects of gilteritinib on HRQoL due to the absence of quality-of-life data for patients who are MRD-positive. pERC concluded that gilteritinib may address an unmet clinical need to a degree that justifies a positive recommendation despite the uncertainty in the clinical value.
• Xospata should only be reimbursed as maintenance treatment for adult patients (aged ≥ 18 years) with newly diagnosed FLT3 ITD–mutated AML who have achieved complete remission with induction therapy, and who are MRD-positive following allo-HCT.
• Xospata should only be reimbursed for a maximum of 24 months, if prescribed by clinicians with expertise in managing patients with AML who have received a stem cell transplant, and if the cost of Xospata is reduced.