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

IDH1 Inhibition Using Ivosidenib as Maintenance Therapy for IDH1-mutant Myeloid Neoplasms Following Allogeneic Stem Cell Transplantation

Phase 1

Completed

Conditions: IDH1 Mutation Myeloid Neoplasms

Interventions: Drug: Ivosidenib

First Posted Date: 2018-06-21

Last Posted Date: 2024-03-08

Lead Sponsor: Massachusetts General Hospital

Target Recruit Count: 18

Registration Number: NCT03564821

Locations: 🇺🇸
Ohio State University Comprehensive Cancer Center, Columbus, Ohio, United States
🇺🇸
Johns Hopkins Cancer Center, Baltimore, Maryland, United States
🇺🇸
Dana Farber Cancer Institute, Boston, Massachusetts, United States

and more 1 locations

IDH1 (AG 120) Inhibitor in Patients With IDH1 Mutated Myelodysplastic Syndrome

Phase 2

Active, not recruiting

Conditions: Acute Myeloid Leukemia
Myelodysplastic Syndromes

Interventions: Drug: AG-120

First Posted Date: 2018-04-19

Last Posted Date: 2025-05-18

Lead Sponsor: Groupe Francophone des Myelodysplasies

Target Recruit Count: 68

Registration Number: NCT03503409

Locations: 🇫🇷
CHU côte de Nacre, Caen, France
🇫🇷
CH Angers, Angers, France
🇫🇷
Centre Hospitalier de la Côte Basque, Bayonne, France

and more 35 locations

A Phase II Randomized Study Comparing the Efficacy and Safety of Targeted Therapy or Cancer Immunotherapy Versus Platinum-Based Chemotherapy in Patients With Cancer of Unknown Primary Site

Phase 2

Completed

Conditions: Cancer of Unknown Primary Site

Interventions: Drug: Alectinib
Drug: Vismodegib
Drug: Carboplatin
Drug: Ipatasertib
Drug: Olaparib
Drug: Erlotinib
Drug: Cisplatin
Drug: Bevacizumab
Drug: Gemcitabine
Drug: Vemurafenib
Drug: Cobimetinib
Drug: Trastuzumab Subcutaneous (SC)
Drug: Pertuzumab
Drug: Atezolizumab
Drug: Paclitaxel
Drug: Entrectinib
Drug: Ivosidenib
Drug: Pemigatinib

First Posted Date: 2018-04-13

Last Posted Date: 2025-02-04

Lead Sponsor: Hoffmann-La Roche

Target Recruit Count: 528

Registration Number: NCT03498521

Locations: 🇫🇷
Centre Antoine Lacassagne, Nice, France
🇫🇷
Institut Curie, Paris, France
🇫🇷
CHU Lyon - Centre Hospitalier Lyon Sud, Pierre-Benite (Lyon), France

and more 127 locations

Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies

Phase 1

Recruiting

Conditions: Hematopoietic and Lymphoid System Neoplasm
Refractory Acute Myeloid Leukemia
Acute Myeloid Leukemia
Myelodysplastic Syndrome
Myeloproliferative Neoplasm
Recurrent Acute Myeloid Leukemia

Interventions: Drug: Azacitidine
Drug: Ivosidenib
Drug: Venetoclax

First Posted Date: 2018-03-20

Last Posted Date: 2025-04-23

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 96

Registration Number: NCT03471260

Locations: 🇺🇸
Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center, Cleveland, Ohio, United States
🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States
🇺🇸
Dana-Farber Cancer Institute, Boston, Massachusetts, United States

and more 1 locations

Study of AG-120 and AG-881 in Subjects With Low Grade Glioma

Phase 1

Completed

Conditions: Glioma

Interventions: Drug: AG-120
Drug: AG881

First Posted Date: 2017-11-17

Last Posted Date: 2025-05-15

Lead Sponsor: Institut de Recherches Internationales Servier

Target Recruit Count: 49

Registration Number: NCT03343197

Locations: 🇺🇸
United States, New York, New York, New York, United States
🇺🇸
United States, California, San Francisco, California, United States
🇺🇸
United States, Massachusetts, Boston, Massachusetts, United States

and more 2 locations

Ivosidenib Expanded Access Program in Relapsed/Refractory AML With an IDH1 Mutation

Conditions: Acute Myeloid Leukemia
Relapsed Adult AML
Relapsed Pediatric AML

First Posted Date: 2017-08-10

Last Posted Date: 2018-07-24

Lead Sponsor: Agios Pharmaceuticals, Inc.

Registration Number: NCT03245424

Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation

Phase 3

Active, not recruiting

Conditions: Newly Diagnosed Acute Myeloid Leukemia (AML)
Untreated AML
AML Arising From Myelodysplastic Syndrome (MDS)
Leukemia, Myeloid, Acute

Interventions: Drug: AG-120
Drug: Placebo
Drug: Azacitidine

First Posted Date: 2017-06-01

Last Posted Date: 2024-08-20

Lead Sponsor: Institut de Recherches Internationales Servier

Target Recruit Count: 146

Registration Number: NCT03173248

Locations: 🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States
🇮🇱
Shamir Medical Center Assaf Harofeh, Tzrifin, Israel
🇺🇸
Norton Cancer Institute - Suburban, Louisville, Kentucky, United States

and more 87 locations

Japanese Bridging Study of Ivosidenib (AG-120) in Healthy Subjects

Phase 1

Completed

Conditions: Healthy

Interventions: Drug: ivosidenib (AG-120)

First Posted Date: 2017-03-07

Last Posted Date: 2018-01-11

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 60

Registration Number: NCT03071770

Locations: 🇺🇸
West Coast Clinical Trial (WCCT), Cypress, California, United States

Study of AG-120 in Previously Treated Advanced Cholangiocarcinoma With IDH1 Mutations (ClarIDHy)

Phase 3

Completed

Conditions: Advanced Cholangiocarcinoma
Metastatic Cholangiocarcinoma

Interventions: Drug: AG-120
Drug: Placebo

First Posted Date: 2016-12-12

Last Posted Date: 2024-08-20

Lead Sponsor: Institut de Recherches Internationales Servier

Target Recruit Count: 187

Registration Number: NCT02989857

Locations: 🇺🇸
University of California, Irvine, Irvine, California, United States
🇺🇸
Northwestern University, Chicago, Illinois, United States
🇺🇸
Dana-Farber Cancer Institute, Boston, Massachusetts, United States

and more 44 locations

Drug-Drug Interaction Study of AG120 in Healthy Subjects

Phase 1

Completed

Conditions: Healthy

Interventions: Drug: AG120
Drug: Itraconazole

First Posted Date: 2016-07-13

Last Posted Date: 2017-06-27

Lead Sponsor: Agios Pharmaceuticals, Inc.

Target Recruit Count: 22

Registration Number: NCT02831972

Drug Development Updates

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