Last reviewed 2026-04-20 · How we verify

Tibsovo (IVOSIDENIB)

Ivosidenib inhibits mutant IDH1, reducing 2-HG levels and promoting myeloid differentiation in AML.

Tibsovo (ivosidenib) is a small molecule isocitrate dehydrogenase 1 (IDH1) inhibitor developed by Agios Pharmaceuticals and currently owned by Servier. It targets the IDH1 enzyme, which is mutated in certain types of cancer, including acute myeloid leukemia and biliary tract cancer. Tibsovo was FDA-approved in 2018 for these indications. The drug is patented and has no generic manufacturers. Key safety considerations include the risk of differentiation syndrome, which can be life-threatening.

At a glance

Mechanism of action

Ivosidenib targets the mutant IDH1 enzyme, which is often found in AML patients. By inhibiting this enzyme, it reduces the levels of 2-HG, a compound that contributes to the disease. This leads to a reduction in cancer cell growth and promotes the normal development of blood cells.

Approved indications

• Acute myeloid leukemia, disease
• Biliary tract cancer

Boxed warnings

• WARNING: DIFFERENTIATION SYNDROME IN AML AND MDS Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal. Symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution [see Warnings and Precautions (5.1) and Adverse Reactions (6.1) ] . WARNING: DIFFERENTIATION SYNDROME IN AML AND MDS See full prescribing information for complete boxed warning. Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution ( 5.1 , 6.1 ).

Common side effects

• leukocytes decreased
• diarrhea
• hemoglobin decreased
• platelets decreased
• glucose increased
• fatigue
• alkaline phosphatase increased
• edema
• potassium decreased
• nausea
• vomiting
• phosphatase decreased

Key clinical trials

• Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial) (PHASE2)
• A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML) (PHASE1)
• Phase II Study Evaluating Ivosidenib Maintenance After SOC Adjuvant Chemotherapy in Curative mIDH1 Cholangiocarcinoma (PHASE2)
• Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies (PHASE1,PHASE2)
• Study Evaluating the Efficacy and Safety of the Addition of Ivosidenib to Oral Azacitidine (Onureg®) in Patients Over 55 With Acute Myeloid Leukemia (AML) and IDH1 Mutation, in Complete Remission After Intensive Chemotherapy. (PHASE2)
• IMPACT-AML: A Randomized Pragmatic Clinical Trial for Relapsed or Refractory Acute Myeloid Leukemia. (PHASE3)
• Ivosidenib as Post-HSCT Maintenance for AML (PHASE2)
• A Study of the Combination of Ivosidenib, Azacitidine, and Venetoclax Followed by Ivosidenib Alone in People With Acute Myeloid Leukemia (PHASE2)

Patents

Primary sources

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