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 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 5/4/2023 | ||
Authorised | 5/4/2023 |
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 |