IDH inhibitors for cholangiocarcinoma – pro
IDHIFA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test. Its relative, ivosidenib is approved for cholangiocarcinoma. On August 25, 2021, the Food and Drug Administration approved ivosidenib (Tibsovo, Servier Pharmaceuticals LLC) for adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.
ClariDHY trial showed that cholangiocarcinoma with IDH1 mutation trials resulted in numerically improved overall survival benefits vs placebo, despite a high rate of crossover. Ivosidenib preserved certain quality of life subscales and was well tolerated.
DHIFA Full Prescribing Information (U.S.). Celgene Corporation. Summit, NJ; July, 2022
enasidenib Prescribing Informaon 2023
A.X. Zhu et al, Final Overall Survival Efficacy Results of Ivosidenib for Patients With Advanced Cholangiocarcinoma With IDH1 Mutation The Phase 3 Randomized Clinical ClarIDHy Trial. JAMA Oncology. September 23, 2021