Protein / target

Isocitrate dehydrogenase [NADP] cytoplasmic

IDH1O75874Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
3
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Isocitrate dehydrogenase (NADP+) activity

Primary system

Endocrine & metabolic

Strongest disease association

Ollier disease

Genetic literature evidence · score 0.84

Therapeutic maturity

Clinically validated target

3 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

3 approved · 4 in clinical development

30 linked trials

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

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.

Drugs targeting this protein

1

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

ivosidenib
Narrow target profileApprovedInhibitor

Isocitrate dehydrogenase [NADP] cytoplasmic inhibitor

Appears in clinical studies involving acute myeloid leukemia, acute myeloid leukemia by FAB classification, cholangiocarcinoma, myelodysplastic syndrome

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

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

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

Ollier disease0.84

Genetic literature · overall 0.52

Maffucci syndrome0.82

Genetic literature · overall 0.63

Enchondromatosis0.79

Genetic · overall 0.63

metaphyseal chondromatosis with D-2-hydroxyglutaric aciduria0.76

Genetic literature · overall 0.55

glioma0.61

Genetic literature · overall 0.64

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

acute myeloid leukemia0.93

Clinical · overall 0.78

oligodendroglioma0.82

Clinical · overall 0.58

cholangiocarcinoma0.80

Clinical · overall 0.65

astrocytoma (excluding glioblastoma)0.79

Clinical · overall 0.59

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

glioblastoma0.66

Literature

Show all associations
acute myeloid leukemia0.78
glioblastoma0.66
cholangiocarcinoma0.65
glioma0.64
Maffucci syndrome0.63
Enchondromatosis0.63
astrocytoma (excluding glioblastoma)0.59
oligodendroglioma0.58
metaphyseal chondromatosis with D-2-hydroxyglutaric aciduria0.55
Ollier disease0.52

Open Targets ranks 827 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 8 total

DS-1001BPhase 2

glioma

BAY1436032Phase 1

acute myeloid leukemia by FAB classification · acute myeloid leukemia

OLUTASIDENIBApproval

acute myeloid leukemia by FAB classification · acute myeloid leukemia · myelodysplastic syndrome

SAFUSIDENIBPhase 3

astrocytoma (excluding glioblastoma) · oligodendroglioma · paraganglioma

IVOSIDENIBApproval

acute myeloid leukemia · acute myeloid leukemia by FAB classification · cholangiocarcinoma

VORASIDENIBApproval

astrocytoma (excluding glioblastoma) · oligodendroglioma · glioma

IDH305Phase 2

paraganglioma · low grade glioma · acute myeloid leukemia

VORASIDENIB CITRATEUnknown

neoplasm

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketAB · GO CC high confPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

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.

Show remaining trials (24)

ACTIVE_NOT_RECRUITING · via ivosidenib · NCT03155620

ClinicalTrials.gov via the drug-target graph.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

acute myeloid leukemiaWell 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 underlying per-type scores are shown above so the calculation can be checked.

What's happening now

3

Recent activity around this target, drawn from one canonical event stream. Every item is reached through a drug that targets this protein, so each event is news about that drug rather than about the protein directly.

  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.