The U.S. Food and Drug Administration (FDA) approved ivosidenib for the treatment of relapsed or refractory (R/R) myelodysplastic syndromes (MDS) in adults with a susceptible IDH1 mutation. The mutation must be detected by an FDA-approved test.
Approval was granted based on the results of a phase I, open-label, single-arm, multicenter trial that found a complete response rate of 38.9%. Results also found common adverse reactions including fatigue, rash, cough, arthralgia, myalgia, and gastrointestinal toxicities such as nausea, diarrhea, constipation, and mucositis.
Prior to approval, ivosidenib received breakthrough therapy designation and priority review from the FDA.
Ivosidenib has also been approved as a treatment for IDH1-mutant R/R acute myeloid leukemia (AML), and both as monotherapy or in combination with azacitidine for adults newly diagnosed with IDH1-AML who are 75 years or older and are ineligible for intensive induction chemotherapy. It is also approved as treatment for IDH1-mutated cholangiocarcinoma.
“The novel use of targeted therapy across IDH-mutated cancers has become a powerful therapeutic option for patients within this molecularly defined subset,” said Amir Fathi, MD, a hematologist at Massachusetts General Hospital in Boston who specializes in myeloid malignancies, in a press release from Servier. “This new indication in IDH1-mutated R/R MDS reinforces the importance of mutational testing to inform treatment decisions and potentially improve patient outcomes.”