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Protein / target

Amidophosphoribosyltransferase

Encoded byPPATQ06203Homo 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

Amidophosphoribosyltransferase

Strongest disease association

Prostate carcinoma

Via encoding gene PPAT · Genetic evidence · score 0.65

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 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.

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.

Approved medicines with mapped indications

1 medicine · 3 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.

Arthritis, Rheumatoid1 medicine
Delayed Graft Function1 medicine
Immune System Diseases1 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.

Azathioprine
Narrow target profileApprovedInhibitor

Amidophosphoribosyltransferase inhibitor

Indicated for Arthritis, Rheumatoid, Delayed Graft Function, Immune System Diseases

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 PPAT

Gene-level evidence surfaced through the gene PPAT 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.

Precursor Cell Lymphoblastic Leukemia-Lymphoma
0.73Moderately supported

Clinical evidence dominant · Open Targets 0.59

Prostate carcinoma
0.67Moderately supported

Genetic evidence dominant · Open Targets 0.41

Prostatic Neoplasms
0.67Moderately supported

Genetic evidence dominant · Open Targets 0.41

View evidence synthesis (3)
Precursor Cell Lymphoblastic Leukemia-LymphomaModerately supported
0.73
agreement 0.580.89
Clinical100%Literature0%

Open Targets aggregate 0.59 · 2 independent evidence families

Prostate carcinomaModerately supported
0.67
agreement 0.530.81
Genetic92%Literature8%

Open Targets aggregate 0.41 · 2 independent evidence families

Prostatic NeoplasmsModerately 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 per-type scores above show the calculation.

Show all associations
Precursor Cell Lymphoblastic Leukemia-Lymphoma0.59
Prostate carcinoma0.41
Prostatic Neoplasms0.41

Drug development

3 compounds recorded · 3 approved

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 (3)
MERCAPTOPURINEApproval
AZATHIOPRINEApproval
AZATHIOPRINE SODIUMApproval

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 Druggable Family support this modality.

Protein degradersEmerging

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

View underlying tractability evidence (4)
SM · Approved DrugSM · Druggable FamilyPR · Database UbiquitinationPR · Half-life Data

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)

NOT_YET_RECRUITING · via Azathioprine · NCT07616154

RECRUITING · via Azathioprine · NCT02939573

ACTIVE_NOT_RECRUITING · via Azathioprine · NCT07044141

COMPLETED · via Azathioprine · NCT03229746

COMPLETED · via Azathioprine · NCT02517684

WITHDRAWN · via Azathioprine · NCT02281799

TERMINATED · via Azathioprine · NCT01880307

ClinicalTrials.gov via the drug-target graph.

What's happening now

12

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. 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 change2008-06-28

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  7. Label change2007-03-06

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  8. Label change2003-11-19

    Label change: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  9. Supplemental approval2002-06-04

    Supplemental approval: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  10. Supplemental approval2001-09-17

    Supplemental approval: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  11. Supplemental approval2001-02-21

    Supplemental approval: AZATHIOPRINE (ANDA075568)

    fda · regulatory · fda · via Azathioprine

  12. Regulatory approval1999-12-13

    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.