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Drug

Ivosidenib

Approved · EMA
ClassIsocitrate dehydrogenase [NADP] cytoplasmic inhibitorEmerging researchLate-stage development

Also known as Tibsovo.

RxNorm2049873ChEMBLCHEMBL3989958ATCL01XM02UNIIQ2PCN8MAM6
1
Research papers
29
Active clinical trials
IDH1
Primary target
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 1b/2, Safety Lead-in and Dose-Expansion, Open Label, Multicenter Trial Investigating the Safety, Tolerability, and Preliminary Activity of Ivosidenib in Combination With Durvalumab and Gemcitabine/Cisplatin as First-line Therapy in Participants With Locally Advanced, Unresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation

Clinical trial2026-07-23Primary completion · ClinicalTrials.gov

A Phase I, Single-Center, Open-Label, Dose De-escalation and Expansion Study Ivosidenib + mFOLFIRINOX in Patients With Resectable Pancreatic Adenocarcinoma

Clinical trial2026-07-15Primary completion · ClinicalTrials.gov

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients With Tumors Harboring IDH1 Mutations

Clinical trial2026-07-01Results posted · ClinicalTrials.gov

Randomized, Sequential, Open-Label Study to Evaluate the Efficacy of IDH Targeted/Non- Targeted Versus Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)

Clinical trial2026-06-23Results expected Q3 2027 · ClinicalTrials.gov

A Phase 1b Multi-center Study of the FLT3 Inhibitor Gilteritinib in Combination With the IDH1 Inhibitor Ivosidenib or the IDH2 Inhibitor Enasidenib for Patients With Relapsed or Refractory Acute Myeloid Leukemia Who Have Co-occurring FLT3/IDH1 or FLT3/IDH2 Mutations

Clinical trial2026-06-22Results expected Q2 2027 · ClinicalTrials.gov

A Randomized, Placebo-Controlled Phase 2 Study of IDH1 Inhibition Using Ivosidenib as Maintenance Therapy for IDH1-mutant Acute Myeloid Leukemia Following Allogeneic Stem Cell Transplantation

Clinical trial2026-06-18Results expected Q1 2028 · ClinicalTrials.gov

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol

Clinical trial2026-06-17Results posted · ClinicalTrials.gov

Phase II Investigator Sponsored Study of CPX-351 in Combination With Ivosidenib for Patients With IDH1 Mutated Acute Myeloid Leukemia or High-Risk MDS

Clinical trial2026-06-12Results expected Q2 2028 · ClinicalTrials.gov

adIVO - A Phase II Trial of Ivosidenib Maintenance After SOC Adjuvant Chemotherapy in Curative mIDH1 Cholangiocarcinoma

Clinical trial2026-06-04Results expected Q4 2030 · ClinicalTrials.gov

Clinical Study of Sonrotoclax Combined With Azacitidine Plus Individualized Targeted Drugs in the Treatment of Newly Diagnosed Adult Acute Myeloid Leukemia

Clinical trial2026-06-03Results expected Q2 2027 · ClinicalTrials.gov

Approval: Tibsovo (EMA)

Regulatory2023-05-04EMA

Final Overall Survival Efficacy Results of Ivosidenib for Patients With Advanced Cholangiocarcinoma With IDH1 Mutation: The Phase 3 Randomized Clinical ClarIDHy Trial.

Research2021-11-01JAMA oncology

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Ivosidenib
Aliases & brands
Tibsovo
RxNorm CUI
2049873
ChEMBL ID
CHEMBL3989958
ATC codes
L01XM02
UNII
Q2PCN8MAM6
Primary mechanism
Isocitrate dehydrogenase [NADP] cytoplasmic inhibitor
Regulatory jurisdictions
ema

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

Isocitrate dehydrogenase [NADP] cytoplasmic
IDH1 · O75874
INHIBITORIsocitrate dehydrogenase [NADP] cytoplasmic inhibitor

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2023-05-04
Latest approval
2023-05-04
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2023-05-04Approval
Approval: Tibsovo (EMA)
Indication: Tibsovo in combination with azacitidine is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1… Show full indication

Tibsovo in combination with azacitidine is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy (see section 5.1). Tibsovo monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

38 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
38
registered trials across all phases
LATEST COMPLETION 2024
29
Active studies
24
Recruiting
6
Late-stage (III+)
4
Completed
5
Discontinued
PHASE DISTRIBUTIONn = 38
Phase 112Phase 1 / 24Phase 216Phase 35Phase N / A1

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled Study of Ivosidenib in Participants ≥18 Years of Age With Locally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation, Untreated or Previously Treated With 1 Systemic Treatment Regimen

RecruitingPH IIIServier Bio-Innovation LLCn=1362030

A Phase 3, Multicenter, Open Label, Randomized, Non-comparative Two-arm Study of Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Adult Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an Isocitrate Dehydrogenase-1 (IDH1) Mutation (PyramIDH Study)

RecruitingPH IIIInstitut de Recherches Internationales Serviern=482028

Molecular Targeted Maintenance Therapy Versus Standard of Care in Advanced Biliary Cancer: an International, Randomised, Controlled, Open-label, Platform Phase 3 Trial

RecruitingPH IIIUNICANCERn=8002028

An Open-Label Early Access Phase 3b Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma

RecruitingPH IIIServier Affaires Médicalesn=2202027

A Single Arm, Open-label Phase 3b Study to Describe the Safety and Tolerability of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy

RecruitingPH IIIServier Affaires Médicalesn=2452027

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase II, Randomized, Active-Controlled, Multi-Center Study Comparing the Efficacy and Safety of Targeted Therapy or Cancer Immunotherapy Guided by Genomic Profiling Versus Platinum-Based Chemotherapy in Patients With Cancer of Unknown Primary Site Who Have Received Three Cycles of Platinum Doublet Chemotherapy

CompletedPH IIHoffmann-La Rochen=5292024

A Phase 1 Study of IDH1 Inhibition Using Ivosidenib as Maintenance Therapy for IDH1-mutant Myeloid Neoplasms Following Allogeneic Stem Cell Transplantation

CompletedPH IMassachusetts General Hospitaln=182023

A Phase 1, Multicenter, Single-Arm Study Evaluating Pharmacokinetic, Pharmacodynamic, Safety, and Clinical Efficacy of Orally Administered Ivosidenib in Chinese Subjects With Relapsed or Refractory Acute Myeloid Leukemia With an IDH1 Mutation

CompletedPH ICStone Pharmaceuticalsn=302023

A Phase 1, Single-Dose, Open-Label Trial to Evaluate the Pharmacokinetics and Safety of AG-120 in Healthy Male Japanese Subjects Relative to Healthy Male Caucasian Subjects

CompletedPH IAgios Pharmaceuticals, Inc.n=602017

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Major research themes2
Bile Duct Neoplasms1Cholangiocarcinoma1
Journals, researchers & institutions
Top journals
  • JAMA oncology1
Leading researchers
  • Abou-Alfa GK1
  • Adeva J1
  • Aguado-Fraile E1
  • Borad MJ1
  • Bridgewater JA1
  • Catenacci DVT1
  • Chamberlain CX1
  • Choe S1
Leading institutions
free-text, unnormalised
  • Dana-Farber Cancer Institute1
  • Helen Diller Family Comprehensive Cancer Center1
  • Hospital Universitario 12 de Octubre1
  • Hospital Universitario Vall d'Hebron1
  • Jiahui International Cancer Center1
  • Johns Hopkins University1

Related drugs

1 matches

Drugs sharing diseases, protein targets and literature with this one. Ranked by graph evidence (shared targets + diseases weighted above co-mentions).

Glycine

Studied across 2 of the same disease areas as Ivosidenib in the shared literature.

2 shared diseases1 shared paper
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.