The Food and Drug Administration (FDA) has granted Priority Review for ivosidenib in the treatment of patients with isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory myelodysplastic syndromes (MDS).
Ivosidenib is an oral IDH1 inhibitor currently marketed under the brand name Tibsovo® for the treatment of newly diagnosed acute myeloid leukemia (AML), relapsed or refractory AML, and locally advanced or metastatic cholangiocarcinoma. The supplemental New Drug Application for the MDS indication is supported by data from a phase 1, open-label study (ClinicalTrials.gov Identifier: NCT02074839) that included 18 patients with IDH1-mutated relapsed or refractory MDS.
Findings showed an objective response rate of 83.3% with 38.9% of patients having a complete response (CR). The median time to CR was 1.87 months (range, 1.0-5.6). At the time of analysis, the median duration of CR had not been reached (range, 1.9-80.8 months) and the median overall survival was 35.7 months (range, 3.7-88.7).
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Among the 9 patients who were dependent on red blood cell and/or platelet transfusions at baseline, 6 (66.7%) became independent of transfusions during any ≥56-day post-baseline period. No new safety signals were reported in the study.
“This filing acceptance and Priority Review for Tibsovo in patients with relapsed or refractory myelodysplastic syndromes underscores our continued work to advance therapeutic progress across IDH mutated cancers, and if approved in this setting, will bring the first and only targeted therapy to patients living with a significant unmet need,” said Susan Pandya, MD, Vice President Clinical Development and Head of Cancer Metabolism Global Development Oncology & Immuno-Oncology, Servier.
This article originally appeared on MPR
