Ivosidenib

Generic Name: Ivosidenib

Brand Names: Tibsovo

Drug Type: Small Molecule

Chemical Formula: C_₂₈H_₂₂ClF_₃N_₆O_₃

CAS Number: 1448347-49-6

Unique Ingredient Identifier: Q2PCN8MAM6

Background: Ivosidenib is a first-in-class isocitrate dehydrogenase-1 (IDH1) inhibitor. IDH1 is an enzyme that is often mutated and overexpressed in some cancers, leading to aberrant cell growth and proliferation. Ivosidenib inhibits mutated IDH1, blocking the enzymatic activity and further differentiation of cancer cells.

Ivosidenib was granted accelerated approval by the FDA in July 2018 for the treatment of relapsed of refractory acute myeloid leukemia in adults. It is currently approved to also treat newly diagnosed acute myeloid leukemia in older adults in combination azacitidine or as monotherapy, as well as locally advanced or metastatic cholangiocarcinoma in adults. The drug is only effective in patients with a susceptible IDH1 mutation.

In February 2023, the EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of ivosidenib and recommended it be granted marketing authorization for the treatment of acute myeloid leukemia and cholangiocarcinoma. It was fully approved by the EMA in May 2023.

Indication: Ivosidenib is an isocitrate dehydrogenase-1 (IDH1) inhibitor approved for use in the US and Europe. It is indicated for the treatment of patients with a susceptible IDH1 mutation with:

Associated Conditions: Acute Myeloid Leukemia, Locally Advanced Cholangiocarcinoma, Metastatic Cholangiocarcinoma, Refractory Acute Myeloid Leukemia (AML), Relapsed Acute Myelogenous Leukemia (AML)

Clinical Trials

An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.

Phase 3

Recruiting

Conditions: Acute Myeloid Leukemia (AML)

Interventions: Drug: Ivosidenib 500mg Oral Tablet
Drug: Azacitidine

First Posted Date: 2023-06-18

Last Posted Date: 2025-04-11

Lead Sponsor: Servier Affaires Médicales

Target Recruit Count: 245

Registration Number: NCT05907057

Locations: 🇦🇹
AKH - Medizinische Universität Wien, Vienna, Austria
🇦🇹
Klinikum Wels-Grieskirchen GmbH, Wels, Austria
🇫🇷
Institut Paoli Calmettes, Marseille, Bouches-du-Rhône, France

and more 12 locations

An Early Access Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma

Phase 3

Recruiting

Conditions: Cholangiocarcinoma

Interventions: Drug: Ivosidenib Oral Tablet

First Posted Date: 2023-05-25

Last Posted Date: 2025-04-20

Lead Sponsor: Servier Affaires Médicales

Target Recruit Count: 220

Registration Number: NCT05876754

Locations: 🇩🇪
Medizinische Fakultaet der Universitaet Freiburg, Freiburg, Germany
🇩🇪
Medizinische Hochschule Hannover, Hannover, Germany
🇩🇪
Klinikum der Universitaet Muenchen-Grosshadern, München, Germany

and more 75 locations

A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML)

Phase 1

Recruiting

Conditions: Acute Myeloid Leukemia (AML)

Interventions: Drug: Gilteritinib
Drug: Ivosidenib
Drug: Enasidenib

First Posted Date: 2023-03-06

Last Posted Date: 2025-03-05

Lead Sponsor: Memorial Sloan Kettering Cancer Center

Target Recruit Count: 36

Registration Number: NCT05756777

Locations: 🇺🇸
Memorial Sloan Kettering Basking Ridge, Basking Ridge, New Jersey, United States
🇺🇸
Memorial Sloan Kettering Monmouth, Middletown, New Jersey, United States
🇺🇸
Memorial Sloan Kettering Bergen, Montvale, New Jersey, United States

and more 4 locations

Personalized Medicine for Advanced Biliary Cancer Patients

Phase 3

Recruiting

Conditions: Biliary Tract Neoplasms

Interventions: Drug: Futibatinib
Drug: Cisplatin
Drug: Ivosidenib
Drug: Gemcitabine
Drug: Zanidatamab
Drug: Trastuzumab
Drug: Neratinib
Drug: Encorafenib
Drug: Binimetinib
Drug: Niraparib

First Posted Date: 2022-11-14

Last Posted Date: 2025-04-23

Lead Sponsor: UNICANCER

Target Recruit Count: 800

Registration Number: NCT05615818

Locations: 🇫🇷
APHP - Hôpital Paul Brousse, Villejuif, France
🇫🇷
Gustave Roussy, Villejuif, France
🇫🇷
APHP - Hôpital Cochin, Paris, France

and more 66 locations

IDH Targeted/Non- Targeted vs Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)

Phase 2

Recruiting

Conditions: Acute Myeloid Leukemia

Interventions: Drug: Azacitidine
Procedure: Biopsy
Drug: Enasidenib
Drug: Ivosidenib
Drug: Venetoclax

First Posted Date: 2022-06-02

Last Posted Date: 2025-05-14

Lead Sponsor: Alice Mims

Target Recruit Count: 125

Registration Number: NCT05401097

Locations: 🇺🇸
Ohio State University Comprehensive Cancer Center, Columbus, Ohio, United States

Ivosidenib + mFOLFIRINOX in Patients With Resectable Pancreatic Adenocarcinoma

Phase 1

Recruiting

Conditions: Pancreatic Ductal Adenocarcinoma
Resectable Pancreatic Cancer

Interventions: Drug: Ivosidenib
Drug: mFOLFIRINOX

First Posted Date: 2022-01-26

Last Posted Date: 2024-07-22

Lead Sponsor: Case Comprehensive Cancer Center

Target Recruit Count: 16

Registration Number: NCT05209074

Locations: 🇺🇸
University Hospitals Cleveland Medical Center, Case Comprehensive Cancer Center, Cleveland, Ohio, United States

Ivosidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH1

Phase 2

Recruiting

Conditions: Clonal Cytopenia of Undetermined Significance

Interventions: Drug: Ivosidenib

First Posted Date: 2021-09-01

Last Posted Date: 2025-02-18

Lead Sponsor: Washington University School of Medicine

Target Recruit Count: 20

Registration Number: NCT05030441

Locations: 🇺🇸
Washington University School of Medicine, Saint Louis, Missouri, United States
🇺🇸
Northwell Health Cancer Institute (R.J. Zuckerberg Cancer Center), Lake Success, New York, United States
🇺🇸
Memorial Sloan Kettering, New York, New York, United States

and more 2 locations