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Protein / target

Isocitrate dehydrogenase [NADP] cytoplasmic

Encoded byIDH1O75874Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
3
Approved medicines
Open Targets target-level
View by indication →
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Isocitrate dehydrogenase (NADP+)

Strongest disease association

Ollier disease

Via encoding gene IDH1 · Genetic literature evidence · score 0.84

Therapeutic position

Established drug target

Small molecules

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function overview

Catalyzes the NADP(+)-dependent oxidative decarboxylation of isocitrate (D-threo-isocitrate) to 2-ketoglutarate (2-oxoglutarate), which is required by other enzymes such as the phytanoyl-CoA dioxygenase.

View complete UniProt function annotation

Catalyzes the NADP(+)-dependent oxidative decarboxylation of isocitrate (D-threo-isocitrate) to 2-ketoglutarate (2-oxoglutarate), which is required by other enzymes such as the phytanoyl-CoA dioxygenase (PubMed:10521434, PubMed:19935646). Plays a critical role in the generation of NADPH, an important cofactor in many biosynthesis pathways (PubMed:10521434). May act as a corneal epithelial crystallin and may be involved in maintaining corneal epithelial transparency (By similarity)

Subcellular location

Cytoplasm, cytosolPeroxisome
Domains and Gene Ontology detail (25)

Gene Ontology

  • Ccytoplasm
  • Ccytosol
  • Cextracellular exosome
  • Cextracellular region
  • Cficolin-1-rich granule lumen
  • Cmitochondrion
  • Cperoxisomal matrix
  • Cperoxisome
  • Csecretory granule lumen
  • Ctertiary granule lumen
  • Fcadherin binding
  • Fidentical protein binding

414 aa · 47 kDa

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Metabolic enzyme activityGO
View supporting evidence

Metabolic enzyme activity

  • ·isocitrate dehydrogenase (NADP+) activity
  • ·2-oxoglutarate metabolic process
  • ·isocitrate metabolic process
  • ·NADP+ metabolic process
View underlying pathways (5)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

ACO1ACO2IDH3GCSOGDHIDH3BIDH3AOGDHLGLUD1MDH2IDH1

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Approved medicines with mapped indications

1 medicine · 2 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Cholangiocarcinoma1 medicine
Leukemia, Myeloid, Acute1 medicine

3 medicines meet Open Targets' target-level approved-medicine definition; the 1 shown here are those on this page with a canonical approved disease indication in the graph. Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

1

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

ivosidenib
Narrow target profileApprovedInhibitor

Isocitrate dehydrogenase [NADP] cytoplasmic inhibitor

Indicated for Cholangiocarcinoma, Leukemia, Myeloid, Acute

Direct interaction with this protein · Only this protein recorded as a target

ChEMBL mechanism, action type, target identity and approved indications. Open Targets clinical status.

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene IDH1

Gene-level evidence surfaced through the gene IDH1 that encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Leukemia, Myeloid, Acute
0.96Well supported

Clinical evidence dominant · Open Targets 0.78

Glioma
0.92Well supported

Clinical evidence dominant · Open Targets 0.64

Cholangiocarcinoma
0.88Well supported

Clinical evidence dominant · Open Targets 0.65

Enchondromatosis
0.87Well supported

Genetic evidence dominant · Open Targets 0.63

Maffucci syndrome
0.83Well supported

Genetic literature evidence dominant · Open Targets 0.63

View evidence synthesis (5)
Leukemia, Myeloid, AcuteWell supported
0.96
agreement 0.881.00
Clinical31%Genetic25%Somatic mutation23%Pathway15%Literature7%

Open Targets aggregate 0.78 · 5 independent evidence families

GliomaWell supported
0.92
agreement 0.831.00
Clinical32%Genetic literature26%Somatic mutation21%Pathway13%Literature8%

Open Targets aggregate 0.64 · 5 independent evidence families

CholangiocarcinomaWell supported
0.88
agreement 0.770.98
Clinical40%Somatic mutation35%Pathway16%Literature9%

Open Targets aggregate 0.65 · 4 independent evidence families

EnchondromatosisWell supported
0.87
agreement 0.760.98
Genetic67%Somatic mutation31%Literature2%Genetic literaturedup

Open Targets aggregate 0.63 · 3 independent evidence families · 1 not counted as duplicate

Maffucci syndromeWell supported
0.83
agreement 0.710.95
Genetic literature55%Somatic mutation39%Literature6%Geneticdup

Open Targets aggregate 0.63 · 3 independent evidence families · 1 not counted as duplicate

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.

Show all associations
Leukemia, Myeloid, Acute0.78
Glioblastoma0.66
Cholangiocarcinoma0.65
Glioma0.64
Maffucci syndrome0.63
Enchondromatosis0.63
Astrocytoma0.59
Oligodendroglioma0.58
metaphyseal chondromatosis with D-2-hydroxyglutaric aciduria0.55
Ollier disease0.52

Drug development

8 compounds recorded · 3 approved · 4 in clinical development · 1 earlier-stage

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 1 drug that targets this protein in Forefront's canonical graph (1 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (8)
DS-1001BPhase 2
BAY1436032Phase 1
OLUTASIDENIBApproval
SAFUSIDENIBPhase 3
IVOSIDENIBApproval
VORASIDENIBApproval
IDH305Phase 2
VORASIDENIB CITRATEUnknown

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small moleculesStrong

Approved Drug and Structure with Ligand support this modality.

AntibodiesEmerging

Feasibility evidence (go cc high conf) — no clinical-stage drug of this modality recorded.

Protein degradersEmerging

Feasibility evidence (database ubiquitination and half-life data) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (8)
SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketAB · GO CC high confPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

View all trials (26)

ACTIVE_NOT_RECRUITING · via ivosidenib · NCT03155620

ClinicalTrials.gov via the drug-target graph.

What's happening now

3

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for a drug that targets this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  1. Trial status changed2026-07-31

    An Open-Label Early Access Phase 3b Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma

    Status changed to Active, not recruiting · ClinicalTrials.gov · via ivosidenib

  2. Regulatory approval2023-05-04

    Approval: Tibsovo (EMA)

    ema · regulatory · ema · via ivosidenib

  3. New publication2021-11-01
    Final Overall Survival Efficacy Results of Ivosidenib for Patients With Advanced Cholangiocarcinoma With IDH1 Mutation: The Phase 3 Randomized Clinical ClarIDHy Trial.

    JAMA oncology · 2021 · 354 citations · Europe PMC · via ivosidenib

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.