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Ivosidenib Oral Tablet
Ivosidenib is an inhibitor of mutant isocitrate dehydrogenase 1 (IDH1) that blocks the production of the oncometabolite 2-hydroxyglutarate, thereby promoting differentiation and apoptosis of IDH1-mutant cancer cells.
Ivosidenib is an inhibitor of mutant isocitrate dehydrogenase 1 (IDH1) that blocks the production of the oncometabolite 2-hydroxyglutarate, thereby promoting differentiation and apoptosis of IDH1-mutant cancer cells. Used for Acute myeloid leukemia (AML) with IDH1 mutation, Cholangiocarcinoma with IDH1 mutation, Other IDH1-mutant solid tumors (in development).
At a glance
| Generic name | Ivosidenib Oral Tablet |
|---|---|
| Sponsor | Servier Bio-Innovation LLC |
| Drug class | IDH1 inhibitor |
| Target | IDH1 (isocitrate dehydrogenase 1, mutant) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
IDH1 mutations are found in certain hematologic malignancies and solid tumors, where they produce elevated levels of 2-hydroxyglutarate, an oncometabolite that drives malignant transformation. By selectively inhibiting mutant IDH1, ivosidenib reduces 2-hydroxyglutarate levels, leading to restoration of normal cellular differentiation pathways and induction of cell death in IDH1-mutant tumors.
Approved indications
- Acute myeloid leukemia (AML) with IDH1 mutation
- Cholangiocarcinoma with IDH1 mutation
- Other IDH1-mutant solid tumors (in development)
Common side effects
- Differentiation syndrome
- Nausea
- Fatigue
- Diarrhea
- Elevated transaminases
- Leukocytosis
Key clinical trials
- 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)
- Ivosidenib as Post-HSCT Maintenance for AML (PHASE2)
- An Early Access Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma (PHASE3)
- A Study of an IDH1m Inhibitor in Participants With IDH1-Mutated Malignancies and Hepatic or Renal Impairment (PHASE1)
- Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation (PHASE3)
- Ivosidenib in Participants With Locally Advanced or Metastatic Conventional Chondrosarcoma Untreated or Previously Treated With 1 Systemic Treatment Regimen (PHASE3)
- Decitabine Alone or in Combination With Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib as Maintenance Therapy for the Treatment of Acute Myeloid Leukemia in Remission (PHASE1)
- Ivosidenib Plus Durvalumab and Gemcitabine/Cisplatin as First-Line Therapy in Participants With Locally Advanced or Metastatic Cholangiocarcinoma With an IDH1 Mutation (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ivosidenib Oral Tablet CI brief — competitive landscape report
- Ivosidenib Oral Tablet updates RSS · CI watch RSS
- Servier Bio-Innovation LLC portfolio CI