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Protein / target

Inosine-5'-monophosphate dehydrogenase 1

Encoded byIMPDH1P20839Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
8
Approved medicines
Open Targets target-level
View by indication →
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

IMP dehydrogenase

Strongest disease association

Retinitis pigmentosa 10

Via encoding gene IMPDH1 · Genetic evidence · score 0.89

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 conversion of inosine 5'-phosphate (IMP) to xanthosine 5'-phosphate (XMP), the first committed and rate-limiting step in the de novo synthesis of guanine nucleotides, and therefore plays an important role in the regulation of cell growth.

View complete UniProt function annotation

Catalyzes the conversion of inosine 5'-phosphate (IMP) to xanthosine 5'-phosphate (XMP), the first committed and rate-limiting step in the de novo synthesis of guanine nucleotides, and therefore plays an important role in the regulation of cell growth. Could also have a single-stranded nucleic acid-binding activity and could play a role in RNA and/or DNA metabolism. It may also have a role in the development of malignancy and the growth progression of some tumors

Subcellular location

CytoplasmNucleus
Domains and Gene Ontology detail (19)

Domains & features

CBS 1CBS 2

Gene Ontology

  • Cazurophil granule lumen
  • Ccytoplasm
  • Ccytosol
  • Cextracellular region
  • Cficolin-1-rich granule lumen
  • Cnucleus
  • Csecretory granule lumen
  • FDNA binding
  • Fidentical protein binding
  • FIMP dehydrogenase activity
  • Fmetal ion binding
  • Fnucleic acid binding

514 aa · 55 kDa · 7 isoforms

Biological roles

Reactome v97

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

View underlying pathways (4)

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.

GMPSIMPDH2ADSS2ATICADSS1AMPD2AMPD3AMPD1ITPAHPRT1IMPDH1

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

4 medicines · 7 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.

Delayed Graft Function1 medicine
Fibrosis1 medicine
Hepatitis C, Chronic1 medicine
Immune System Diseases1 medicine
Liver Cirrhosis1 medicine
Virus Diseases1 medicine
Neoplasms1 medicine

8 medicines meet Open Targets' target-level approved-medicine definition; the 4 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

4

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.

Ribavirin
Narrow target profileApprovedInhibitor

Inosine-5'-monophosphate dehydrogenase 1 inhibitor

Indicated for Fibrosis, Hepatitis C, Chronic, Liver Cirrhosis, Virus Diseases

Direct interaction with this protein · 1 of 2 recorded protein targets — narrow recorded profile

Mycophenolic Acid
ApprovedInhibitor

Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor

Indicated for Immune System Diseases

Acts on a complex — shared with IMPDH2 · 1 of 2 recorded protein targets — narrow recorded profile

mycophenolate mofetil
ApprovedInhibitor

Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor

Indicated for Delayed Graft Function

Acts on a complex — shared with IMPDH2 · 1 of 2 recorded protein targets — narrow recorded profile

thioguanine
ApprovedInhibitor

Inosine-5'-monophosphate dehydrogenase (IMPDH) inhibitor

Indicated for Neoplasms

Acts on a complex — shared with IMPDH2 · 1 of 2 recorded protein targets — narrow recorded profile

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 IMPDH1

Gene-level evidence surfaced through the gene IMPDH1 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.

Retinitis pigmentosa 10
0.91Well supported

Genetic evidence dominant · Open Targets 0.68

Retinitis Pigmentosa
0.88Well supported

Genetic evidence dominant · Open Targets 0.73

Leber congenital amaurosis 11
0.79Well supported

Genetic evidence dominant · Open Targets 0.60

Leber Congenital Amaurosis
0.70Moderately supported

Genetic literature evidence dominant · Open Targets 0.63

Precursor Cell Lymphoblastic Leukemia-Lymphoma
0.65Moderately supported

Clinical evidence dominant · Open Targets 0.53

View evidence synthesis (5)
Retinitis pigmentosa 10Well supported
0.91
agreement 0.791.00
Genetic84%Animal model14%Literature2%Genetic literaturedup

Open Targets aggregate 0.68 · 3 independent evidence families · 1 not counted as duplicate

Retinitis PigmentosaWell supported
0.88
agreement 0.760.99
Genetic68%Animal model30%Literature2%Genetic literaturedup

Open Targets aggregate 0.73 · 3 independent evidence families · 1 not counted as duplicate

Leber congenital amaurosis 11Well supported
0.79
agreement 0.670.91
Genetic84%Animal model15%Literature1%Genetic literaturedup

Open Targets aggregate 0.60 · 3 independent evidence families · 1 not counted as duplicate

Leber Congenital AmaurosisModerately supported
0.70
agreement 0.570.82
Genetic literature68%Animal model30%Literature3%Geneticdup

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

Precursor Cell Lymphoblastic Leukemia-LymphomaModerately supported
0.65
agreement 0.500.81
Clinical100%Literature1%

Open Targets aggregate 0.53 · 2 independent evidence families

This ranking differs from Open Targets' own: re-weighting moves genetically-evidenced diseases above more heavily co-mentioned ones. 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
Retinitis Pigmentosa0.73
Retinitis pigmentosa 100.68
Leber Congenital Amaurosis0.63
Leber congenital amaurosis 110.60
Precursor Cell Lymphoblastic Leukemia-Lymphoma0.53

Drug development

9 compounds recorded · 8 approved · 1 in clinical development

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 4 drugs that target this protein in Forefront's canonical graph (4 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (9)
MYCOPHENOLATE MOFETIL HYDROCHLORIDEApproval
MYCOPHENOLIC ACIDApproval
MYCOPHENOLATE MOFETILApproval
MIZORIBINEApproval
MYCOPHENOLATE SODIUMApproval
RIBAVIRINApproval
AVN-944Phase 2
THIOGUANINEApproval
MERIMEPODIBApproval

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 (9)
SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · Med-Quality PocketSM · Druggable FamilyAB · GO CC high confPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

leukopeniaClinPGxgastrointestinal intoleranceClinPGxacute rejection after kidney transplantationClinPGx

Terms indexed against this target in Open Targets' safety data, with their datasource. These are annotations, not causal claims: the direction of effect (whether activation or inhibition is implicated), species and evidence strength are not captured here, so an entry does not mean that modulating this target is known to cause that condition.

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 mycophenolate mofetil · NCT07634536

RECRUITING · via mycophenolate mofetil · NCT07566377

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 3 drugs that target 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. Product recall2026-08-26

    Recall (Class II): MYCOPHENOLATE MOFETIL

    fda · safety · fda · via mycophenolate mofetil

  2. Trial status changed2026-08-19

    A Prospective Study of Optimal Cord Selection for Haplo-Cord Transplantation: Targeting the Inherited Paternal Antigen (IPA) and Matching for the Non-Inherited Maternal Antigen (NIMA)

    Status changed to Completed · ClinicalTrials.gov · via mycophenolate mofetil

  3. Supplemental approval2026-07-29

    Supplemental approval: MYCOPHENILIC ACID (ANDA091248)

    fda · regulatory · fda · via Mycophenolic Acid

  4. Supplemental approval2026-07-29

    Supplemental approval: MYCOPHENOLATE MOFETIL (ANDA065379)

    fda · regulatory · fda · via mycophenolate mofetil

  5. Supplemental approval2026-07-29

    Supplemental approval: MYCOPHENOLATE MOFETIL (ANDA065410)

    fda · regulatory · fda · via mycophenolate mofetil

  6. Product recall2026-07-02

    Recall (Class II): MYCOPHENOLATE MOFETIL

    fda · safety · fda · via mycophenolate mofetil

  7. Regulatory approval2026-06-01

    Approval: MYCOPHENOLATE MOFETIL (ANDA218566)

    fda · regulatory · fda · via mycophenolate mofetil

  8. New publication2017-08-10
    Safety and efficacy of a fixed-dose combination regimen of grazoprevir, ruzasvir, and uprifosbuvir with or without ribavirin in participants with and without cirrhosis with chronic hepatitis C virus genotype 1, 2, or 3 infection (C-CREST-1 and C-CREST-2, part B): two randomised, phase 2, open-label trials.

    The lancet. Gastroenterology & hepatology · 2017 · 12 citations · Europe PMC · via Ribavirin

  9. New publication2015-11-16
    Sofosbuvir and Velpatasvir for HCV in Patients with Decompensated Cirrhosis.

    The New England journal of medicine · 2015 · 586 citations · Europe PMC · via Ribavirin

  10. Safety communication2014-12-11

    Drug Safety Update: Mycophenolate mofetil: pure red cell aplasia

    mhra · safety · mhra · via mycophenolate mofetil

  11. New publication2014-05-04
    Sofosbuvir and ribavirin in HCV genotypes 2 and 3.

    The New England journal of medicine · 2014 · 578 citations · Europe PMC · via Ribavirin

  12. New publication2014-04-10
    Retreatment of HCV with ABT-450/r-ombitasvir and dasabuvir with ribavirin.

    The New England journal of medicine · 2014 · 399 citations · Europe PMC · via Ribavirin

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.