Overview
Olutasidenib (FT-2102) is a selective and potent isocitrate dehydrogenase-1 (IDH1) inhibitor approved by the FDA in December 2022. It is indicated for the treatment of relapsed or refractory acute myeloid leukemia (AML) in patients with a susceptible IDH1 mutation as determined by an FDA-approved test. IDH1 mutations are common in different types of cancer, such as gliomas, AML, intrahepatic cholangiocarcinoma, chondrosarcoma, and myelodysplastic syndromes (MDS), and they lead to an increase in 2-hydroxyglutarate (2-HG), a metabolite that participates in tumerogenesis. Olutasidenib inhibits the mutated IDH1 specifically, and provides a therapeutic benefit in IDH1-mutated cancers. Other IDH1 inhibitors, such as ivosidenib, have also been approved for the treatment of relapsed or refractory AML. Olutasidenib is orally bioavailable and capable of penetrating the blood-brain barrier, and is also being evaluated for the treatment of myelodysplastic syndrome (MDS), as well as solid tumors and gliomas (NCT03684811).
Indication
Olutasidenib is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.
Associated Conditions
- Refractory Acute Myeloid Leukemia (AML)
- Relapsed Acute Myelogenous Leukemia (AML)
Research Report
Olutasidenib (Rezlidhia): A Comprehensive Monograph on the Mutant IDH1 Inhibitor for Relapsed/Refractory Acute Myeloid Leukemia
Executive Summary
Olutasidenib, marketed under the brand name Rezlidhia, is an orally administered, potent, and selective small-molecule inhibitor of mutated isocitrate dehydrogenase-1 (mIDH1).[1] It represents a significant advancement in the targeted therapy of acute myeloid leukemia (AML). The drug's mechanism of action is precisely targeted, inhibiting the neomorphic enzymatic activity of mIDH1, which is responsible for the oncogenic production of 2-hydroxyglutarate (2-HG). By suppressing 2-HG, Olutasidenib alleviates the epigenetic blockade characteristic of mIDH1-driven malignancies, thereby restoring normal hematopoietic cell differentiation.[4]
The clinical efficacy of Olutasidenib was definitively established in the pivotal cohort of the Phase 2 study 2102-HEM-101, which evaluated its use in adult patients with relapsed or refractory (R/R) AML harboring a susceptible IDH1 mutation. The trial met its primary endpoint, demonstrating a composite complete remission (CR) and CR with partial hematologic recovery (CRh) rate of 35%. The most striking feature of this response was its unprecedented durability, with a median duration of 25.9 months—a substantial improvement over existing therapies in this patient population.[6] This sustained effect suggests a profound and lasting impact on the underlying disease biology, potentially altering the natural history of the disease for responding patients.
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/09/04 | Not Applicable | Not yet recruiting | |||
2025/06/24 | Phase 2 | Recruiting | |||
2025/01/20 | Phase 2 | Recruiting | Justin Watts, MD | ||
2024/10/31 | Phase 2 | Recruiting | |||
2024/09/19 | Phase 2 | Recruiting | |||
2024/08/22 | Phase 2 | Recruiting | |||
2024/08/09 | Phase 1 | Recruiting | City of Hope Medical Center | ||
2024/06/06 | Phase 1 | Recruiting | |||
2023/12/08 | Phase 2 | Recruiting | |||
2022/10/03 | Phase 2 | Recruiting |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Rigel Pharmaceuticals, Inc. | 71332-005 | ORAL | 150 mg in 1 1 | 12/8/2022 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
| No EMA approvals found for this drug. | |||
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 |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
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 |
|---|---|---|---|---|---|
| No TGA approvals found for this drug. | |||||
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
|---|---|---|---|---|---|
| No Health Canada approvals found for this drug. | |||||
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
|---|---|---|---|---|---|
| No CIMA AEMPS (Spain) approvals found for this drug. | |||||
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Philippines FDA approvals found for this drug. | |||||
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Saudi SFDA approvals found for this drug. | |||||
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Malaysia NPRA approvals found for this drug. | |||||
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
|---|---|---|---|---|---|
| No UK EMC drug information found for this drug. | |||||
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