Enasidenib

Overview

Enasidenib is an orally available treatment for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with specific mutations in the isocitrate dehydrogenase 2 (IDH2) gene, which is a recurrent mutation detected in 12-20% of adult patients with AML . Patients eligible for this treatment are selected by testing the presence of IDH2 mutations in the blood or bone marrow. This small molecule acts as an allosteric inhibitor of mutant IDH2 enzyme to prevent cell growth, and it also has shown to block several other enzymes that play a role in abnormal cell differentiation. First developed by Agios Pharmaceuticals and licensed to Celgene, enasidenib was approved by U.S. Food and Drug Administration on August 1, 2017.

Indication

Indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation.

Associated Conditions

Refractory Acute Myeloid Leukemia (AML)
Relapsed Acute Myelogenous Leukemia (AML)

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
A Study of Enasidenib in People With T-Cell Lymphoma	2025/01/02	NCT06756308	Phase 2	Recruiting	Memorial Sloan Kettering Cancer Center
Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial)	2024/11/04	NCT06672146	Phase 2	Recruiting	National Cancer Institute (NCI)
Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)	2024/08/29	NCT06577441	Phase 2	Recruiting	National Cancer Institute (NCI)
Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2A Decentralized Trial	2024/02/05	NCT06240754	Phase 2	Recruiting	Washington University School of Medicine
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors	2023/12/20	NCT06176989	Phase 2	Recruiting	National Cancer Institute (NCI)
A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML)	2023/03/06	NCT05756777	Phase 1	Recruiting	Memorial Sloan Kettering Cancer Center
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)	2022/10/03	NCT05564390	Phase 2	Recruiting	National Cancer Institute (NCI)
Enasidenib in Combination With Cobimetinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia	2022/07/01	NCT05441514	Phase 1	Suspended	City of Hope Medical Center
IDH Targeted/Non- Targeted vs Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)	2022/06/02	NCT05401097	Phase 2	Recruiting	Alice Mims
Enasidenib in MDS &Non-proliferative Chronic Myelomonocytic Leukemia w/o IDH2 Mutation	2022/03/16	NCT05282459	Phase 1	Completed	Stanford University

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Idhifa	Celgene Corporation	59572-710	ORAL	100 mg in 1 1	11/30/2020
Idhifa	Celgene Corporation	59572-705	ORAL	50 mg in 1 1	11/30/2020

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.

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