Idhifa

(Enasidenib)

ApprovedSmall molecule

KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Enasidenib is used to treat a type of blood cancer called acute myeloid leukemia (AML) in adults. It works by targeting a specific mutation in cancer cells that helps stop their growth.[2] Idhifa is currently approved and in use across 8 indications[4].[1]

Idhifa is one of 176 small molecule programs targeting Chondrosarcoma. It is currently in Approved.

Indications

Total Trials

Active Trials

175

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

ChondrosarcomaRecruiting

Hematopoietic and Lymphoid Cell NeoplasmApproved

Liver DiseaseApproved

Acute Myeloid LeukemiaRecruiting

Clonal Cytopenia of Undetermined SignificanceRecruiting

Pediatric CancerRecruiting

T - Cell LymphomaActive, not recruiting

Acute Myeloid Leukemia by FAB ClassificationApproved

ChondrosarcomaPhase 2

Recruiting

Hematopoietic and Lymphoid Cell NeoplasmApproved

Approved

Liver DiseaseApproved

Approved

Acute Myeloid LeukemiaPhase 2

Recruiting

Clonal Cytopenia of Undetermined SignificancePhase 2

Recruiting

Pediatric CancerPreclinical

Recruiting

T - Cell LymphomaPhase 2

Active, not recruiting

Acute Myeloid Leukemia by FAB ClassificationApproved

Approved

Competition

Competitive Landscape

235 competitors in Acute Myeloid Leukemia

#DrugCompanyPhaseTrials

Idamycin

Pfizer

Phase 3

60 2

Novantrone

Pfizer

Phase 3

34 3

Idhifa

KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Approved

9 4

Vepesid

AAvyxa

Phase 3

52 5

Vanflyta

Daiichi Sankyo

Phase 3

16 6

Cerubidine

HHikma Pharmaceuticals LLC

Phase 3

42 7

Cerubidine

HHikma Pharmaceuticals LLC

Phase 3

32 8

Neupogen

EEastern Cooperative Oncology Group

Phase 3

30 9

Nexavar

Cellcept

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 1Recruiting

Acute Myeloid Leukemia

NCT05010772

Q1 2027

Phase 2Recruiting

Acute Myeloid Leukemia

NCT03383575

Phase 2Recruiting

Myelodysplastic Syndrome

NCT06577441

Phase 2Recruiting

Acute Myeloid Leukemia

NCT06672146

Q2 2027

Phase 1Active, not recruiting

Acute Myeloid Leukemia (AML)

NCT05756777

Q4 2027

Phase 1/2Recruiting

Acute Myeloid Leukemia

NCT04774393

Clinical

Trials by Indication[1]

Acute Myeloid Leukemia9 trials

A Phase 1b Master Trial to Investigate CPX-351 in Subjects With Previously Untreated Acute Myeloid Leukemia

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 1·NCT04075747·57 enrolled

Enasidenib in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia With an IDH2 Gene Mutation

Withdrawn·RRoswell Park Cancer Institute·Phase 2·NCT03881735

Decitabine/Cedazuridine and Venetoclax in Combination With Ivosidenib or Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

Recruiting·MM.D. Anderson Cancer Center·Phase 1/2·NCT04774393·84 enrolled

Show 6 more trials

Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With an IDH2 Mutation

Active, not recruiting·CChildren's Oncology Group·Phase 2·NCT04203316·1 enrolled

Master Screening and Reassessment Protocol (MSRP) for the NCI MyeloMATCH Clinical Trials

Recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT05564390·2,000 enrolled

A Randomized Phase II Trial of ASTX727 and Venetoclax Compared With ASTX727, Venetoclax, and Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy

Recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT06672146·93 enrolled

Oral Decitabine-Based Maintenance Therapy in Patients With AML in Remission

Recruiting·MM.D. Anderson Cancer Center·Phase 1·NCT05010772·125 enrolled

Targeted Therapy With the IDH2-Inhibitor Enasidenib (AG221) for High-Risk IDH2-Mutant Myelodysplastic Syndrome

Recruiting·MM.D. Anderson Cancer Center·Phase 2·NCT03383575·63 enrolled

Randomized, Sequential, Open-Label Study to Evaluate the Efficacy of IDH Targeted/Non- Targeted Versus Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)

Recruiting·AAlice Mims·Phase 2·NCT05401097·125 enrolled

Hepatic Impairment2 trials

A Study to Assess the Pharmacokinetics of Enasidenib (CC-90007) in Subjects With Moderate and Severe Hepatic Impairment.

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 1·NCT03290443·31 enrolled

Pharmacokinetics of Enasidenib (CC-90007) in Participants With Mild, Moderate and Severe Hepatic Impairment

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 1·NCT04573582·40 enrolled

Clonal Cytopenia of Undetermined Significance2 trials

A Study of Enasidenib in People With Clonal Cytopenia of Undetermined Significance

Completed·MMemorial Sloan Kettering Cancer Center·Phase 1·NCT05102370·4 enrolled

A Pilot Study of Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2: A Decentralized Trial

Recruiting·WWashington University School of Medicine·Phase 2·NCT06240754·15 enrolled

Myeloid Neoplasm2 trials

IDH2 Inhibition Using Enasidenib as Maintenance Therapy for IDH2-mutant Myeloid Neoplasms Following Allogeneic Stem Cell Transplantation

Completed·MMassachusetts General Hospital·Phase 1·NCT03515512·23 enrolled

IDH2-Post-Allo-Trial for Patients with IDH2-mut Myeloid Neoplasms After Allo-SCT

Completed·HHeinrich-Heine University, Duesseldorf·Phase 2·NCT04522895·50 enrolled

Advanced Hematologic Malignancies1 trial

Phase 1/2 Study of Enasidenib (AG-221) in Adults With Advanced Hematologic Malignancies With an Isocitrate Dehydrogenase Isoform 2 (IDH2) Mutation

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 1/2·NCT01915498·345 enrolled

12 more indications

Trial1 trial

Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial

Terminated·OOHSU Knight Cancer Institute·Phase 1·NCT03878524·2 enrolled

Angioimmunoblastic T-Cell Lymphoma1 trial

Study of Orally Administered Enasidenib (AG-221) in Adults With Advanced Solid Tumors, Including Glioma, or Angioimmunoblastic T-cell Lymphoma, With an IDH2 Mutation

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 1/2·NCT02273739·21 enrolled

Blood Cancer1 trial

Study of Enasidenib and Venetoclax in IDH2-Mutated Blood Cancers

Terminated·UUniversity Health Network, Toronto·Phase 1/2·NCT04092179·27 enrolled

Myeloproliferative Neoplasms1 trial

A Study of Fedratinib With IDH Inhibition in Advanced-Phase, IDH-Mutated Ph-Negative Myeloproliferative Neoplasms

Withdrawn·UUniversity of Chicago·Phase 1·NCT04955938

Adult1 trial

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

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 1·NCT04310527·28 enrolled

Drug Interactions1 trial

A Study of Perpetrator Drug Interactions of Enasidenib in AML Patients

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 1·NCT03720366·40 enrolled

Myelofibrosis1 trial

Combined Ruxolitinib and Enasidenib in Patients With Accelerated/Blast-phase Myeloproliferative Neoplasm or Chronic-phase Myelofibrosis With an IDH2 Mutation

Completed·JJohn Mascarenhas·Phase 2·NCT04281498·6 enrolled

Myelodysplastic Syndrome1 trial

A Randomized Phase II Trial of Enasidenib-Based Therapies Versus Cedazuridine-Decitabine in Higher-Risk IDH2-Mutated Myelodysplastic Syndrome: A MyeloMATCH Sub-Study

Recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT06577441·54 enrolled

Chondrosarcoma1 trial

Phase II Study of Enasidenib in IDH2-mutated Malignant Sinonasal and Skull Base Tumors

Recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT06176989·40 enrolled

Acute Myeloid Leukemia (AML)1 trial

A Phase 1b Multi-center Study of the FLT3 Inhibitor Gilteritinib in Combination With the IDH1 Inhibitor Ivosidenib or the IDH2 Inhibitor Enasidenib for Patients With Relapsed or Refractory Acute Myeloid Leukemia Who Have Co-occurring FLT3/IDH1 or FLT3/IDH2 Mutations

Active, not recruiting·MMemorial Sloan Kettering Cancer Center·Phase 1·NCT05756777·8 enrolled

T - Cell Lymphoma1 trial

A Phase 2 Study of Enasidenib in IDH2-Mutant Angioimmunoblastic T-Cell Lymphoma

Active, not recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT06756308·2 enrolled

Pediatric Cancer1 trial

European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors

Recruiting·GGustave Roussy, Cancer Campus, Grand Paris·Preclinical·NCT02813135·472 enrolled

Regulatory

FDA Approval Timeline[5]

Aug 1, 2017

IDHIFA

NDA NDA209606

Aug 1, 2017

IDHIFA

NDA NDA209606

Sep 27, 2019

IDHIFA

NDA NDA209606

Nov 24, 2020

IDHIFA

NDA NDA209606

Jul 26, 2021

IDHIFA

NDA NDA209606

Dec 21, 2023

IDHIFA

NDA NDA209606

Dec 18, 2024

IDHIFA

NDA NDA209606

Jan 16, 2025

IDHIFA

NDA NDA209606

Sources

References

[1]
ClinicalTrials.gov — 28 trial records for Enasidenib. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Idhifa drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Enasidenib compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Enasidenib. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA209606). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Idhifa. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Karuna Therapeutics, Inc., a Bristol Myers Squibb company company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Isocitrate Dehydrogenase [NADP], Mitochondrial Inhibitor[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Patent Landscape[6]

Patent Cliff

Jan 7, 2033

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9512107Jan 7, 2033

10294215Jan 7, 2033

10093654Aug 1, 2034

9738625Aug 1, 2034

9732062Sep 16, 2034

Idhifa

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2027

Q2 2027

Q4 2027

Trials by Indication[1]

Acute Myeloid Leukemia9 trials

Hepatic Impairment2 trials

Clonal Cytopenia of Undetermined Significance2 trials

Myeloid Neoplasm2 trials

Advanced Hematologic Malignancies1 trial

Trial1 trial

Angioimmunoblastic T-Cell Lymphoma1 trial

Blood Cancer1 trial

Myeloproliferative Neoplasms1 trial

Adult1 trial

Drug Interactions1 trial

Myelofibrosis1 trial

Myelodysplastic Syndrome1 trial

Chondrosarcoma1 trial

Acute Myeloid Leukemia (AML)1 trial

T - Cell Lymphoma1 trial

Pediatric Cancer1 trial

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

Patent Landscape[6]

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