Clinical Efficacy in IDH1-Mutant Malignancies

TIBSOVO inhibits mutant IDH1, reducing 2-hydroxyglutarate levels and inducing differentiation in myeloid malignancies. In newly diagnosed AML (combined with azacitidine), it improved median overall survival (24 vs. 7.9 months) and complete remission (CR) rates (47% vs. 15%). For relapsed/refractory AML, 32.8% achieved CR/CRh, with median duration of 8.2 months. In MDS, 38.9% achieved CR, and in cholangiocarcinoma, progression-free survival doubled (HR: 0.37). Efficacy is contingent on confirmed IDH1 mutations (e.g., R132C/H) via FDA-approved tests. Transfusion independence rates were 37–67% across indications.