Drug · Small Molecule

Ivosidenib

CI-DRUG-00000313Explore in graph →NCIt C114383 CHEMBL3989958 CIViC Approved

Regulatory

Approvals (9)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

emaRegulatory9 approval records from 2 sources (hover a row badge for its dataset) · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
EU EMA	—	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.	approved	May 4, 2023	ema
US FDA	—	Efficacy supplement 2023-10-24 (see label)	approved	Oct 24, 2023	openfda
US FDA	—	Efficacy supplement 2022-05-25 (see label)	approved	May 25, 2022	openfda
US FDA	—	Efficacy supplement 2021-08-25 (see label)

Data updated 2 days agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 3	33	22	7	44	9	EU, US	Jul 20, 2018	Mar 1, 2014
Leukemia	Approved	Phase 3	20	15	3	23	1	US	Jul 20, 2018	Mar 1, 2014
Malignant Central Nervous System Neoplasm	Phase2	Phase 2	2	0	0	2	0	—	—	Jul 31, 2017
Malignant Liver Neoplasm	Phase2	Phase 2	2	0	0	2	0

Curated evidence

Clinical evidence (16)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–16 of 16 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
IDH1 Mutation3
Ivosidenib	Acute Myeloid Leukemia

Clinical trials

Trials with this intervention (46)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–46 of 46 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT03839771	A Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy, Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myedysplastic Syndrome EB2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapy(HOVON150AML)	active not recruiting	Phase 3	968	Stichting Hemato-Oncologie voor Volwassenen NederlandOTHER	15	Sep 22, 2026	clinicaltrials
NCT07507760

Molecule

Record

Kind: Small Molecule
Mechanism: Isocitrate dehydrogenase [NADP] cytoplasmic inhibitor
Targets: 1 gene
UNII: Q2PCN8MAM6
Aliases: AG 120, AG-120, AG120, Ivosidenib, Tibsovo, TIBSOVO

Data updated 21 hours agoSource updated unknown

Identifiers

Codes (2)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01XM02	Antineoplastic agents
EMA product	EMEA/H/C/005936	Tibsovo

Regulatory status is jurisdiction-specific and time-bound. Nothing on this page is a treatment recommendation.

IDH1 R1327
Ivosidenib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	3	submitted	EID13076 In a subgroup analysis of a multicenter phase I study, 17 patients with relapsed or refractory IDH1 R132-mutated acute myeloid leukemia who responded to ivosidenib 500 mg once daily subsequently under… (full text at CIViC) PMID 33772143 · DiNardo et al., 2021 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	4	submitted	EID13077 In a multicenter phase I study, 33 patients with newly diagnosed IDH1 R132-mutated acute myeloid leukemia who were ineligible for standard therapy received ivosidenib 500 mg once daily. The CR+CRh rat… (full text at CIViC) PMID 31841594 · Roboz et al., 2020 · Open in CIViC	civic
Azacitidine + IvosidenibCombination	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	A	Supports Sensitivity Response	5	submitted	EID12899 In the double-blind, placebo-controlled, phase III AGILE trial (NCT03173248), 146 patients with newly diagnosed IDH1-mutated acute myeloid leukemia (AML) who were ineligible for intensive induction ch… (full text at CIViC) PMID 35443108 · Montesinos et al., 2022 · Open in CIViC	civic
〃	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	A	Supports Sensitivity Response	5	submitted	EID12901 This post hoc long-term follow-up analysis of the phase 3, double-blind, placebo-controlled AGILE trial (NCT03173248) reports updated outcomes for 148 patients with newly diagnosed IDH1-mutated AML in… (full text at CIViC) PMID 40706052 · Montesinos et al., 2025 · Open in CIViC	civic
Ivosidenib	Cholangiocarcinoma	Predictive	A	Supports Sensitivity Response	5	accepted	EID10886 The multicenter randomized double-blind phase 3 study included patients with IDH1-mutant cholangiocarcinoma who had progressed on previous therapy. Patients were randomly assigned to Ivosidenib or pla… (full text at CIViC) PMID 32416072 · Abou-Alfa et al., 2020 · Open in CIViC	civic
〃	Chondrosarcoma	Predictive	A	Supports Sensitivity Response	2	submitted	EID11194 In a phase 1 study (NCT02073994), 21 patients with advanced IDH-mutant chondrosarcoma received the IDH1-mutant specific inhibitor ivosidenib. Plasma levels of the 2-HG oncometabolite decreased to nor… (full text at CIViC) PMID 32208957 · Tap et al., 2020 · Open in CIViC	civic
〃	Myelodysplastic SyndromeCURATED_BROADER	Predictive	B	Supports Sensitivity Response	3	submitted	EID13287 In the relapsed or refractory myelodysplastic syndrome (MDS) substudy of the phase 1, open-label, single-arm, multinational first-in-human trial of ivosidenib (NCT02074839), adults with MDS carrying a… (full text at CIViC) PMID 38640348 · DiNardo et al., 2024 · Open in CIViC	civic

IDH1 R132H2
Ivosidenib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	3	submitted	EID13075 In a multicenter phase I dose-escalation and expansion study, 125 patients with relapsed or refractory IDH1-mutated acute myeloid leukemia (AML) received ivosidenib 500 mg once daily. The complete rem… (full text at CIViC) PMID 29860938 · DiNardo et al., 2018 · Open in CIViC	civic
〃	Pancreatic Ductal Adenocarcinoma	Predictive	C	Does Not Support Sensitivity Response	2	accepted	EID5987 A 48-year-old female was diagnosed with metastatic pancreatic ductal adenocarcinoma and subsequently started on standard of care chemotherapy, during which her hepatic lesions progressed. Detailed mol… (full text at CIViC) PMID 27466707 · Brody et al., 2018 · Open in CIViC	civic