IDHIFA (enasidenib mesylate)
IDHIFA treats adults with acute myeloid leukemia (AML) that has returned or has not responded to previous therapies. It helps patients whose cancer cells carry a specific mutation in the isocitrate dehydrogenase-2 (IDH2) enzyme. Before starting this medication, patients must undergo a specialized test to confirm they have this specific genetic marker.
How IDHIFA Works
This drug works by blocking the activity of the mutated IDH2 enzyme, which is found in certain types of leukemia cells. By inhibiting this enzyme, the medication lowers levels of a substance called 2-hydroxyglutarate and encourages immature cancer cells to develop into mature, functioning myeloid cells. This process helps reduce the overall number of leukemia blast cells in the blood.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2017-08-01
- Patent Cliff
- 2034
- Routes
- ORAL
- Dosage Forms
- TABLET
IDHIFA Approval History
What IDHIFA Treats
1 indicationsIDHIFA is approved for 1 conditions since its original approval in 2017. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Acute Myeloid Leukemia
IDHIFA Boxed Warning
DIFFERENTIATION SYNDROME Patients treated with IDHIFA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, acute respiratory distress, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, lymphadenopathy, bone pain, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom reso...
WARNING: DIFFERENTIATION SYNDROME Patients treated with IDHIFA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, acute respiratory distress, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, lymphadenopathy, bone pain, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution [see Warnings and Precautions (5.1) and Adverse Reactions (6.1) ] . WARNING: DIFFERENTIATION SYNDROME See full prescribing information for complete boxed warning. Patients treated with IDHIFA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution ( 5.1 , 6.1 ).
IDHIFA Target & Pathway
ProTarget
IDHIFA Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in IDHIFA's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications IDHIFA treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to IDHIFA
3 of 18FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
33 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT06672146 | NCI-2024-08951 NCI-2024-08951, MM1OA-S03 | Ph 2 | recruiting | Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial) |
| NCT05401097 | OSU-21330 NCI-2022-01324, R01CA262496 | Ph 2 | recruiting | 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) |
| NCT05564390 | NCI-2022-07006 NCI-2022-07006, MYELOMATCH | Ph 2 | recruiting | MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) |
| NCT06577441 | NCI-2024-07049 NCI-2024-07049, MM1MDS-A01 | Ph 2 | recruiting | 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) |
| NCT05441514 | 21751 NCI-2022-04926, 21751 | Ph 1 | completed | Enasidenib in Combination With Cobimetinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia |
| NCT06176989 | 10001552 001552-C | Ph 2 | recruiting | Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors |
| NCT06756308 | 23-269 | Ph 2 | recruiting | A Study of Enasidenib in People With T-Cell Lymphoma |
| NCT05756777 | 22-174 | Ph 1 | recruiting | A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML) |
| NCT03728335 | 18117 NCI-2018-02371, 18117 | Ph 2 | recruiting | Enasidenib (AG-221) Maintenance Post Allogeneic HCT in Patients With IDH2 Mutation |
| NCT03683433 | 2018-0499 NCI-2018-01919, 2018-0499 | Ph 2 | recruiting | Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation |
| NCT03383575 | 2016-0981 NCI-2018-00987, 2016-0981 | Ph 2 | recruiting | Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome |
| NCT06240754 | 202405007 | Ph 2 | recruiting | Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2A Decentralized Trial |
| NCT05102370 | 21-268 | Ph 1 | completed | A Study of Enasidenib in People With Clonal Cytopenia of Undetermined Significance |
| NCT02813135 ESMART | 2016-000133-40 2016/2396, 2024-514791-40-00 | Ph 1, Ph 2 | recruiting | European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors |
| NCT05010772 | 2021-0237 NCI-2021-08496, 2021-0237 | Ph 1 | recruiting | Decitabine Alone or in Combination With Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib as Maintenance Therapy for the Treatment of Acute Myeloid Leukemia in Remission |
| NCT04203316 | ADVL18P1 NCI-2019-07902, ADVL18P1 | Ph 2 | active not recruiting | Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With an IDH2 Mutation |
| NCT04774393 | 2020-1220 NCI-2021-00893, 2020-1220 | Ph 1, Ph 2 | recruiting | Decitabine/Cedazuridine and Venetoclax in Combination With Ivosidenib or Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia |
| NCT03515512 | 18-022 | Ph 1 | completed | IDH2 Inhibition Using Enasidenib as Maintenance Therapy for IDH2-mutant Myeloid Neoplasms Following Allogeneic Stem Cell Transplantation |
| NCT04281498 results posted | GCO 07-0548-0008 P01CA108671, MPN-RC 119 | Ph 2 | completed | Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation |
| NCT01915498 results posted | AG-221-C-001 2013-001784-23 | Ph 1, Ph 2 | completed | Phase 1/2 Study of Enasidenib (AG-221) in Adults With Advanced Hematologic Malignancies With an Isocitrate Dehydrogenase Isoform 2 (IDH2) Mutation |
| NCT04522895 | AG-221CL-AML-PI13299 2019-001416-30 | Ph 2 | completed | IDH2-Post-Allo-Trial for Patients with IDH2-mut Myeloid Neoplasms After Allo-SCT |
| NCT04955938 | IRB21-0483 | Ph 1 | withdrawn | A Study of Fedratinib With IDH Inhibition in Advanced-Phase, IDH-Mutated Ph-Negative Myeloproliferative Neoplasms |
| NCT04573582 | CC-90007-CP-007 U1111-1257-6808 | Ph 1 | completed | Pharmacokinetics of Enasidenib (CC-90007) in Participants With Mild, Moderate and Severe Hepatic Impairment |
| NCT03878524 | STUDY00015588 NCI-2020-02743, STUDY00015588 | Ph 1 | terminated | Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial |
| NCT03720366 | CC-90007-CP-004 U1111-1218-1974 | Ph 1 | completed | A Study of Perpetrator Drug Interactions of Enasidenib in AML Patients |
| NCT04092179 | 19-5939 ENAVEN-AML | Ph 1, Ph 2 | terminated | Study of Enasidenib and Venetoclax in IDH2-Mutated Blood Cancers |
| NCT04075747 V-FAST | JZP025-101 | Ph 1 | completed | A Phase 1b Master Trial to Investigate CPX-351 in Subjects With Previously Untreated Acute Myeloid Leukemia |
| NCT03825796 | 18-001416 NCI-2018-02998 | Ph 2 | active not recruiting | CPX-351 Plus Enasidenib for Relapsed AML |
| NCT04655391 | 20456 NCI-2020-10595, 20456 | Ph 1 | withdrawn | Glasdegib-Based Treatment Combinations for the Treatment of Patients With Relapsed Acute Myeloid Leukemia Who Have Undergone Hematopoietic Cell Transplantation |
| NCT03881735 | I 67118 NCI-2019-00332, I 67118 | Ph 2 | withdrawn | Enasidenib in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia With an IDH2 Gene Mutation |
| NCT02273739 results posted | AG221-C-003 2014-003424-47 | Ph 1, Ph 2 | completed | Study of Orally Administered Enasidenib (AG-221) in Adults With Advanced Solid Tumors, Including Glioma, or Angioimmunoblastic T-cell Lymphoma, With an IDH2 Mutation |
| NCT03290443 | CC-90007-CP-003 U1111-1202-3186 | Ph 1 | completed | A Study to Assess the Pharmacokinetics of Enasidenib (CC-90007) in Subjects With Moderate and Severe Hepatic Impairment. |
| NCT04310527 | CC-90007-CP-006 U1111-1243-9124 | Ph 1 | completed | Bioavailability of Enasidenib (CC-90007) Sprinkle Formulation Relative to the Reference Tablet Formulation and Effect of Food on the Pharmacokinetics of Sprinkle Formulation in Healthy Adults |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
IDHIFA FDA Label Details
Indications & Usage
FDA Label (PDF)IDHIFA is indicated for the treatment of Acute Myeloid Leukemia.
WARNING: DIFFERENTIATION SYNDROME Patients treated with IDHIFA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, acute respiratory distress, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peri...
IDHIFA Patents & Exclusivity
Patents (6 active)
Pro Intelligence Preview
Deep insights for IDHIFA
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2034
- • 24 active patents
Trial Analysis
- • 34 total trials
- • Stage: Growth
Competitive Landscape
- • 18 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment