Protein / target

Amidophosphoribosyltransferase

PPATQ06203Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
3
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Amidophosphoribosyltransferase activity

Strongest disease association

prostate carcinoma

Genetic evidence · score 0.65

Therapeutic maturity

Clinically validated target

3 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

3 approved

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 formation of phosphoribosylamine from phosphoribosylpyrophosphate (PRPP) and glutamine

Domains and Gene Ontology detail (11)

Domains & features

Glutamine amidotransferase type-2

Gene Ontology

  • Ccytosol
  • F4 iron, 4 sulfur cluster binding
  • Famidophosphoribosyltransferase activity
  • Fmetal ion binding
  • P'de novo' AMP biosynthetic process
  • P'de novo' IMP biosynthetic process
  • P'de novo' XMP biosynthetic process
  • PGMP biosynthetic process
  • Ppurine nucleobase biosynthetic process
  • Ppurine nucleotide biosynthetic process

517 aa · 57 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

  • ·amidophosphoribosyltransferase activity
View underlying pathways (1)

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.

GARTPAICSADSLATICCADPFASPRPS2PRPS1GLULPRPS1L1PPAT

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.

Azathioprine
Narrow target profileApprovedInhibitor

Amidophosphoribosyltransferase inhibitor

Appears in clinical studies involving rheumatoid arthritis, Crohn disease, ulcerative colitis, immune system disorder

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.

prostate carcinoma0.65

Genetic · overall 0.41

prostate cancer0.65

Genetic · overall 0.41

Highest-confidence therapeutic associations

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

acute lymphoblastic leukemia0.98

Clinical · overall 0.59

rheumatoid arthritis0.97

Clinical · overall 0.59

Crohn disease0.95

Clinical · overall 0.58

ulcerative colitis0.92

Clinical · overall 0.56

lymphoid leukemia0.73

Clinical · overall 0.44

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

lymphoblastic lymphoma0.40

Clinical

lupus nephritis0.39

Clinical

acute promyelocytic leukemia0.39

Clinical

Show all associations
acute lymphoblastic leukemia0.59
rheumatoid arthritis0.59
Crohn disease0.58
ulcerative colitis0.56
lymphoid leukemia0.44
prostate carcinoma0.41
prostate cancer0.41
lymphoblastic lymphoma0.40
lupus nephritis0.39
acute promyelocytic leukemia0.39

Open Targets ranks 1,175 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 · 3 total

MERCAPTOPURINEApproval

acute lymphoblastic leukemia · acute lymphoblastic leukemia · lymphoid leukemia

AZATHIOPRINEApproval

rheumatoid arthritis · Crohn disease · ulcerative colitis

AZATHIOPRINE SODIUMApproval

Tractability

SM · Approved DrugSM · Druggable FamilyPR · Database UbiquitinationPR · Half-life Data

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)

WITHDRAWN · via Azathioprine · NCT02281799

TERMINATED · via Azathioprine · NCT01880307

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 lymphoblastic leukemiaModerately supported
0.73
agreement 0.580.89
Clinical100%Literature0%

Open Targets aggregate 0.59 · 2 independent evidence families

rheumatoid arthritisModerately supported
0.73
agreement 0.560.89
Clinical100%

Open Targets aggregate 0.59 · 1 independent evidence family

Crohn diseaseModerately supported
0.71
agreement 0.550.88
Clinical100%

Open Targets aggregate 0.58 · 1 independent evidence family

ulcerative colitisModerately supported
0.69
agreement 0.530.86
Clinical100%

Open Targets aggregate 0.56 · 1 independent evidence family

prostate carcinomaModerately supported
0.67
agreement 0.530.81
Genetic92%Literature8%

Open Targets aggregate 0.41 · 2 independent evidence families

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

14

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. Label change2026-07-16

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  2. New publication2023-10-17
    The Genetics of Inflammatory Bowel Disease.

    Molecular diagnosis & therapy · 2024 · 69 citations · Europe PMC · via Azathioprine

  3. Regulatory approval2021-06-21

    Approval: Jayempi (EMA)

    ema · regulatory · ema · via Azathioprine

  4. Label change2019-04-23

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  5. Label change2015-05-29

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  6. Label change2011-03-23

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  7. Label change2008-06-28

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  8. Label change2007-03-06

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  9. Label change2003-11-19

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  10. Supplemental approval2002-06-04

    Supplemental approval: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  11. Supplemental approval2001-09-17

    Supplemental approval: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  12. Supplemental approval2001-02-21

    Supplemental approval: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

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.