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.

openfdaRegulatory8 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
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)	approved	Aug 25, 2021	openfda
US FDA	—	Efficacy supplement 2019-05-02 (see label)	approved	May 2, 2019	openfda
US

Data updated 7 hours 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 10 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 R1327

Ivosidenib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

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

Supports Sensitivity Response

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

Supports Sensitivity Response

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

Supports Sensitivity Response

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

Supports Sensitivity Response

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

Supports Sensitivity Response

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

Supports Sensitivity Response

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 R132C1
Ivosidenib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	2	accepted	EID2331 In a clinical study of 17 relapsed or refractory AML patients harboring IDH1 mutations, these patients were associated with response to AG-120 monotherapy; 4/7 patients who achieved a response experie… (full text at CIViC) PMID 25583779 · 2015 · Open in CIViC	civic

IDH1 R132C1

Ivosidenib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

accepted

EID2331

In a clinical study of 17 relapsed or refractory AML patients harboring IDH1 mutations, these patients were associated with response to AG-120 monotherapy; 4/7 patients who achieved a response experie… (full text at CIViC)

PMID 25583779 · 2015 · Open in CIViC

civic

IDH1 R132C + IDH1 R132H1
Ivosidenib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	accepted	EID12903 Popovici-Muller et al. reported preliminary efficacy data from an ongoing phase 1 study of ivosidenib (AG-120) monotherapy in patients with IDH1-mutated malignancies. In patients with relapsed or refr… (full text at CIViC) PMID 29670690 · Popovici-Muller et al., 2018 · Open in CIViC	civic

IDH1 R132C + IDH1 R132H1

Ivosidenib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

accepted

EID12903

Popovici-Muller et al. reported preliminary efficacy data from an ongoing phase 1 study of ivosidenib (AG-120) monotherapy in patients with IDH1-mutated malignancies. In patients with relapsed or refr… (full text at CIViC)

PMID 29670690 · Popovici-Muller et al., 2018 · 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

IDH1 R132H2

Ivosidenib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

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

Does Not Support Sensitivity Response

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

IDH1 R132L1
Ivosidenib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	—	rejected	EID2021 In a clinical study of 17 relapsed or refractory AML patients harboring IDH1 mutations, patients were associated with response to AG-120 monotherapy; 4/7 patients who achieved a response experienced c… (full text at CIViC) PMID 25583779 · 2015 · Open in CIViC	civic

IDH1 R132L1

Ivosidenib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

—

rejected

EID2021

In a clinical study of 17 relapsed or refractory AML patients harboring IDH1 mutations, patients were associated with response to AG-120 monotherapy; 4/7 patients who achieved a response experienced c… (full text at CIViC)

PMID 25583779 · 2015 · Open in CIViC

civic

IDH1 R132S1
Ivosidenib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	B	Supports Sensitivity Response	—	submitted	EID2340 In a clinical study of 17 relapsed or refractory AML patients harboring IDH1 mutations, patients were associated with response to AG-120 monotherapy; 4/7 patients who achieved a response experienced c… (full text at CIViC) PMID 25583779 · 2015 · Open in CIViC	civic

IDH1 R132S1

Ivosidenib

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

—

submitted

EID2340

PMID 25583779 · 2015 · Open in CIViC

civic