Initial US Approval
- 20211
Indications
- in combination with azacitidine or as monotherapy for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.1
- the treatment of adult patients with relapsed or refractory AML with a susceptible IDH1 mutation as detected by an FDA-approved test.1
- The treatment of adult patients with relapsed or refractory myelodysplastic syndromes (MDS) with a susceptible IDH1 mutation as detected by an FDA-approved test.1
- The treatment of adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma (CCA) with an IDH1 mutation as detected by an FDA-approved test.1
Recommended Dose/Route
- Ivosidenib, 500 mg, taken orally once daily until disease progression or unacceptable toxicity.1
Pivotal Study
- AG120-C-005 (NCT02989857)2
- Key Inclusion Criteria: Patients with histologically confirmed, advanced, IDH1-mutant CCA who had progressed on previous therapy, and had up to 2 previous treatment regimens for advanced disease, an Eastern Cooperative Oncology Group performance status score of 0 or 1, and a measurable lesion as defined by Response Evaluation Criteria in Solid Tumors version 1.1.2
- Treatment: Patients were administered 500 mg of ivosidenib or a placebo orally once daily in continuous 28-day cycles (allowing for a variation of plus or minus 2 days), beginning on day 1 of cycle 1. Study visits were scheduled biweekly during cycles 1 through 3 (on days 1 and 15) and on day 1 of subsequent cycles. Treatment continued until disease progression as determined by the investigator, the emergence of unacceptable toxicity, confirmed pregnancy, death, withdrawal of consent, loss to follow-up, or unblinding or termination of the study.2
Safety and Adverse Event (AE) Management
- Common adverse reactions (≥10%): The most frequently reported any grade AEs were fatigue (43%), nausea (41%), diarrhea (35%), abdominal pain (35%), cough (27%), decreased appetite (24%), ascites (23%), vomiting (23%), anemia (18%), rash (15%), headache (13%), peripheral neuropathy (11%), and QT prolongation (10%).1
- Common laboratory abnormalities (≥10%): The most frequently reported laboratory abnormalities were decreased hemoglobin (40%), elevated AST (34%), and elevated bilirubin (30%).1
- Dose reductions due to AEs: 3%1
- Permanent discontinuation due to AEs: 2%1
References
- TIBSOVO (ivosidenib). Prescribing information. Servier Pharmaceuticals LLC; 2023. https://www.tibsovopro.com/pdf/prescribinginformation.pdf
- Abou-Alfa GK, Macarulla T, Javle MM, et al. Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study. Lancet Oncol. 2020;21(6):796-807. doi:10.1016/S1470-2045(20)30157-1