Ivosidenib
- Generic Name
- Ivosidenib
- Brand Names
- Tibsovo
- Drug Type
- Small Molecule
- Chemical Formula
- C28H22ClF3N6O3
- 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)
A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapy
- Conditions
- Leukemia, Myeloid, Acute
- Interventions
- First Posted Date
- 2016-02-09
- Last Posted Date
- 2025-04-23
- Lead Sponsor
- Celgene
- Target Recruit Count
- 130
- Registration Number
- NCT02677922
- Locations
- 🇮🇹
Local Institution - 250, Pesaro, Italy
🇪🇸Local Institution - 381, Madrid, Spain
🇪🇸Local Institution - 380, Malaga, Spain
Safety Study of AG-120 or AG-221 in Combination With Induction and Consolidation Therapy in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) With an IDH1 and/or IDH2 Mutation
- Conditions
- Newly Diagnosed Acute Myeloid Leukemia (AML)AML Arising After Exposure to Genotoxic InjuryUntreated AMLAML Arising From Myelodysplastic Syndrome (MDS)AML Arising From Antecedent Hematologic Disorder (AHD)
- Interventions
- First Posted Date
- 2015-12-17
- Last Posted Date
- 2025-04-11
- Lead Sponsor
- Institut de Recherches Internationales Servier
- Target Recruit Count
- 153
- Registration Number
- NCT02632708
- Locations
- 🇺🇸
UCLA Medical Center, Los Angeles, California, United States
🇺🇸Hackensack University Medical Center, Hackensack, New Jersey, United States
🇺🇸Johns Hopkins University, Baltimore, Maryland, United States
Food Effect Study of AG120 in Healthy Subjects
- First Posted Date
- 2015-10-20
- Last Posted Date
- 2016-11-29
- Lead Sponsor
- Agios Pharmaceuticals, Inc.
- Target Recruit Count
- 36
- Registration Number
- NCT02579707
- Locations
- 🇺🇸
Research Site, Madison, Wisconsin, United States
Study of Orally Administered AG-120 in Subjects With Advanced Solid Tumors, Including Glioma, With an IDH1 Mutation
- Conditions
- GliomaCholangiocarcinomaChondrosarcomaOther Advanced Solid Tumors
- Interventions
- First Posted Date
- 2014-02-28
- Last Posted Date
- 2025-01-27
- Lead Sponsor
- Institut de Recherches Internationales Servier
- Target Recruit Count
- 174
- Registration Number
- NCT02073994
Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation
- Conditions
- Myelodysplastic SyndromesRelapsed or Refractory Acute Myeloid Leukemia (AML)Untreated AMLOther IDH1-mutated Positive Hematologic Malignancies
- Interventions
- First Posted Date
- 2014-02-28
- Last Posted Date
- 2024-07-25
- Lead Sponsor
- Institut de Recherches Internationales Servier
- Target Recruit Count
- 291
- Registration Number
- NCT02074839
- Locations
- 🇺🇸
John Hopkins Cancer Center, Baltimore, Maryland, United States
🇺🇸UT Southwestern Medical Center, Dallas, Texas, United States
🇺🇸Massachusetts General Hospital, Boston, Massachusetts, United States
Drug Development Updates
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