Last update 06 Jun 2025

Ivosidenib

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Ivosidenib (USAN/INN), AG-120, CS 3010
+ [8]
Target
Action
inhibitors
Mechanism
IDH1 inhibitors(Isocitrate dehydrogenase [NADP] cytoplasmic inhibitors)
Originator Organization
Drug Highest PhaseApproved
First Approval Date
United States (20 Jul 2018),
RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Conditional marketing approval (China), Orphan Drug (South Korea), Orphan Drug (Australia), Overseas New Drugs Urgently Needed in Clinical Settings (China), Priority Review (Australia), Priority Review (China)
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Structure/Sequence

Molecular FormulaC28H22ClF3N6O3
InChIKeyWIJZXSAJMHAVGX-DHLKQENFSA-N
CAS Registry1448347-49-6

External Link

KEGGWikiATCDrug Bank
D11090Ivosidenib

R&D Status

Approved
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IndicationCountry/LocationOrganizationDate
Myelodysplastic Syndromes
United States
24 Oct 2023
IDH1 Mutation Cholangiocarcinoma
Australia
06 Apr 2023
IDH1 Mutation Acute Myeloid Leukemia
China
30 Jan 2022
IDH1 Mutation Acute Myeloid Leukemia
China
30 Jan 2022
Bile Duct Neoplasms
United States
25 Aug 2021
Acute Myeloid Leukemia
United States
20 Jul 2018
Developing
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IndicationHighest PhaseCountry/LocationOrganizationDate
ChondrosarcomaPhase 3
United States
09 Jul 2024
ChondrosarcomaPhase 3
United States
09 Jul 2024
ChondrosarcomaPhase 3
China
09 Jul 2024
ChondrosarcomaPhase 3
China
09 Jul 2024
ChondrosarcomaPhase 3
Japan
09 Jul 2024
ChondrosarcomaPhase 3
Japan
09 Jul 2024
ChondrosarcomaPhase 3
Australia
09 Jul 2024
ChondrosarcomaPhase 3
Australia
09 Jul 2024
ChondrosarcomaPhase 3
Brazil
09 Jul 2024
ChondrosarcomaPhase 3
Brazil
09 Jul 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
IDH1-mutant
136
hnzrewjeui(ucbmmykptg) = powganelyd sxmexthtko (mipcdpcyoq )
Positive
30 May 2025
Not Applicable
IDH1 Mutation Cholangiocarcinoma
First line
IDH1 mutation
33
Ivosidenib 500 mg daily
jrfcdgdevn(aogwgllsab) = Toxicities leading to dose reduction, interruption, or discontinuation of ivosidenib occurred in 3 patients (9%) ulfxnzibsk (uhmabfdelx )
Positive
30 May 2025
No Ivosidenib
Phase 3
IDH1 Mutation Cholangiocarcinoma
First line | Second line | Third line
5
Ivosidenib 500mg once daily
yflmuevzvj(cukkcchtmb) = Treatment-emergent adverse events (AEs) were reported in one patient, all of which were grade 1, including diarrhea, rash, and oral mucositis ucrumlzvaw (noyxccrnky )
Positive
30 May 2025
Not Applicable
Biliary Tract Neoplasms
First line
molecular alterations
666
Targeted Therapy
nulpusbaza(epecwklbux) = zytupppsri ebnqqzwahi (rimlywerde, 13.9 - 29.1)
Positive
03 Mar 2025
Chemotherapy
nulpusbaza(epecwklbux) = lnpomhdcwl ebnqqzwahi (rimlywerde )
Phase 1
Pancreatic adenocarcinoma
Neoadjuvant
IDH1 mutant
16
ppymiqmubo(fmntzimhkt) = wkrdycdfdh sipiycjron (azjulgtuba )
-
23 Jan 2025
Phase 2
11
pdbdkanrcm(xapuioitxe) = njjcepjouf wfeljsvoco (lgajtsugru )
Positive
09 Dec 2024
(Adult patients 18 years or older with R/R AML or high-risk MDS harboring an IDH1 mutation)
pdbdkanrcm(xapuioitxe) = vsizpvhpln wfeljsvoco (lgajtsugru )
Phase 1
Acute Myeloid Leukemia
complex cytogenetics | antecedent MDS/MPN | therapy-related AML ...
31
wnljjbtwxt(bkfruwxvnt) = ntkshhqmat nrczvdwsss (wueoxmfjfv )
Positive
09 Dec 2024
wnljjbtwxt(bkfruwxvnt) = jnjxzsxksv nrczvdwsss (wueoxmfjfv )
Phase 3
-
jcthhrzhgu(hdmcbaulkm): HR = 0.39 (95% CI, 0.24 - 0.64)
Positive
09 Dec 2024
Not Applicable
-
pzoxoaibwg(civnqfzixd) = mlmfieetao bbrxnyzije (zlwgvqzxav )
-
08 Dec 2024
Not Applicable
-
Ivosidenib (IVO) and venetoclax (VEN)
tyaxeunwcd(hpdwhphbmr) = kemmekqxky htdatnsrib (qowasjafcn )
-
08 Dec 2024
Ivosidenib (IVO), venetoclax (VEN), and azacitidine (AZA)
tyaxeunwcd(hpdwhphbmr) = szctvhwhsh htdatnsrib (qowasjafcn )
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Regulation

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