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Ivosidenib

Generic Name
Ivosidenib
Brand Names
Tibsovo
Drug Type
Small Molecule
Chemical Formula
C28H22ClF3N6O3
CAS Number
1448347-49-6
Unique Ingredient Identifier
Q2PCN8MAM6

Overview

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.

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

Title
Posted
Study ID
Phase
Status
Sponsor
2025/06/05
Phase 1
Not yet recruiting
2024/12/05
Phase 2
Recruiting
2024/11/27
Phase 2
Not yet recruiting
2024/09/12
Phase 2
Not yet recruiting
2024/07/15
Phase 1
Recruiting
2024/06/20
Phase 3
Recruiting
2024/03/04
Phase 1
Recruiting
2023/12/26
N/A
Recruiting
2024/06/10
Phase 3
Recruiting
2023/10/13
Phase 2
Active, not recruiting

FDA Drug Approvals

Approved Product
Manufacturer
NDC Code
Route
Strength
Effective Date
Servier Pharmaceutical LLC
72694-617
ORAL
250 mg in 1 1
10/30/2023
Agios Pharmaceuticals, Inc.
71334-100
ORAL
250 mg in 1 1
5/3/2019

HSA Drug Approvals

Approved Product
Manufacturer
Approval Number
Dosage Form
Strength
Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product
Company
Approval Number
Drug Type
Dosage Form
Approval Date
Ivosidenib Tablets
国药准字HJ20220002
化学药品
片剂
1/30/2022

PPB Drug Approvals

Approved Product
Registration No.
Company
Licence No.
Strength
Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product
ARTG ID
Sponsor
Registration Type
Status
Registration Date
TIBSOVO ivosidenib 250 mg film-coated tablet bottle
391874
Medicine
A
4/6/2023
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