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Protein / target

3-hydroxy-3-methylglutaryl-coenzyme A reductase

Encoded byHMGCRP04035Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
15
Approved medicines
Open Targets target-level
View by indication →
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Hydroxymethylglutaryl-CoA reductase (NADPH)

Strongest disease association

Hyperlipidemias

Via encoding gene HMGCR · Genetic evidence · score 0.82

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 (3S)-hydroxy-3-methylglutaryl-CoA (HMG-CoA) to mevalonic acid, the rate-limiting step in the synthesis of cholesterol and other isoprenoids, thus plays a critical role in cellular cholesterol homeostasis.

View complete UniProt function annotation

Catalyzes the conversion of (3S)-hydroxy-3-methylglutaryl-CoA (HMG-CoA) to mevalonic acid, the rate-limiting step in the synthesis of cholesterol and other isoprenoids, thus plays a critical role in cellular cholesterol homeostasis (PubMed:21357570, PubMed:2991281, PubMed:36745799, PubMed:6995544). HMGCR is the main target of statins, a class of cholesterol-lowering drugs (PubMed:11349148, PubMed:18540668, PubMed:36745799)

Subcellular location

Endoplasmic reticulum membranePeroxisome membrane
Domains and Gene Ontology detail (22)

Domains & features

SSD

Gene Ontology

  • Ccytoplasmic side of endoplasmic reticulum membrane
  • Cendoplasmic reticulum
  • Cendoplasmic reticulum membrane
  • Cperoxisomal membrane
  • Fcoenzyme A binding
  • FGTPase regulator activity
  • Fhydroxymethylglutaryl-CoA reductase (NADPH) activity
  • FNADPH binding
  • Pcholesterol biosynthetic process
  • Pcholesterol biosynthetic process via desmosterol
  • Pcholesterol biosynthetic process via lathosterol
  • Pcoenzyme A metabolic process

888 aa · 97 kDa · 3 isoforms

Biological roles

Reactome v97

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

Lipid & lipoprotein metabolismUniProt · GO · ReactomeTranscriptional regulationReactome
View supporting evidence

Lipid & lipoprotein metabolism

  • ·Catalyzes the conversion of (3S)-hydroxy-3-methylglutaryl-CoA (HMG-CoA) to mevalonic aci…
  • ·cholesterol biosynthetic process
  • ·cholesterol biosynthetic process via desmosterol
  • ·cholesterol biosynthetic process via lathosterol

Transcriptional regulation

  • ·PPARA activates gene expression
  • ·Activation of gene expression by SREBF (SREBP)
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.

INSIG1MVKHMGCS1HMGCS2INSIG2SREBF2SQLEFDPSSREBF1FDFT1HMGCR

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 · 9 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.

Coronary Artery Disease1 medicine
Coronary Disease1 medicine
Dyslipidemias1 medicine
Hypercholesterolemia1 medicine
Hyperlipidemias1 medicine
Hyperlipoproteinemia Type II1 medicine
Myocardial Infarction1 medicine
Stroke1 medicine
Broader indication categories (1)
Cardiovascular Diseases1 medicine

Broad umbrella indications (e.g. “Neoplasms”). Shown here because every medicine also appears under a more specific disease above — kept for completeness, de-emphasised for clarity.

15 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.

Simvastatin
Narrow target profileApprovedInhibitor

HMG-CoA reductase inhibitor

Indicated for Coronary Artery Disease, Coronary Disease, Dyslipidemias, Hypercholesterolemia

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 HMGCR

Gene-level evidence surfaced through the gene HMGCRthat 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.

Hyperlipidemias
0.96Well supported

Genetic evidence dominant · Open Targets 0.73

Coronary Artery Disease
0.94Well supported

Clinical evidence dominant · Open Targets 0.72

Familial hypercholesterolemia
0.92Well supported

Clinical evidence dominant · Open Targets 0.70

Stroke
0.87Well supported

Clinical evidence dominant · Open Targets 0.68

Cardiovascular Diseases
0.86Well supported

Clinical evidence dominant · Open Targets 0.67

View evidence synthesis (5)
HyperlipidemiasWell supported
0.96
agreement 0.851.00
Genetic51%Clinical47%Literature3%

Open Targets aggregate 0.73 · 3 independent evidence families

Coronary Artery DiseaseWell supported
0.94
agreement 0.841.00
Clinical46%Genetic42%Animal model6%Literature6%

Open Targets aggregate 0.72 · 4 independent evidence families

Familial hypercholesterolemiaWell supported
0.92
agreement 0.821.00
Clinical50%Genetic41%Animal model7%Literature2%

Open Targets aggregate 0.70 · 4 independent evidence families

StrokeWell supported
0.87
agreement 0.770.98
Clinical59%Genetic38%Literature3%

Open Targets aggregate 0.68 · 3 independent evidence families

Cardiovascular DiseasesWell supported
0.86
agreement 0.750.97
Clinical60%Genetic38%Literature2%

Open Targets aggregate 0.67 · 3 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
Hyperlipidemias0.73
Coronary Artery Disease0.72
Familial hypercholesterolemia0.70
Stroke0.68
Cardiovascular Diseases0.67
Muscular dystrophy, limb-girdle, autosomal recessive 280.66
Diabetes Mellitus, Type 20.63
Myocardial Infarction0.62
Angina Pectoris0.61

Drug development

16 compounds recorded · 15 approved · 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 (10)
PRAVASTATINApproval
ROSUVASTATIN CALCIUMApproval
LOVASTATINApproval
PITAVASTATINApproval
ATORVASTATIN CALCIUMApproval
PITAVASTATIN CALCIUMApproval
PITAVASTATIN SODIUMApproval
ATORVASTATINApproval
PITAVASTATIN MAGNESIUMApproval
CERIVASTATINApproval

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 (uniprot sigp or tmhmm) — no clinical-stage drug of this modality recorded.

Protein degradersEmerging

Feasibility evidence (literature and uniprot ubiquitination) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (10)
SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · UniProt SigP or TMHMMPR · LiteraturePR · UniProt UbiquitinationPR · Database UbiquitinationPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

Decrease, FertilityAOP-Wiki

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)

UNKNOWN · via Simvastatin · NCT00842920

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 change2026-08-05

    Label change: SIMVASTATIN (ANDA076685)

    fda · regulatory · fda · via Simvastatin

  2. New publication2017-06-07
    Effect of high-dose simvastatin on cognitive, neuropsychiatric, and health-related quality-of-life measures in secondary progressive multiple sclerosis: secondary analyses from the MS-STAT randomised, placebo-controlled trial.

    The Lancet. Neurology · 2017 · 88 citations · Europe PMC · via Simvastatin

  3. New publication2015-09-01
    Achievement of dual low-density lipoprotein cholesterol and high-sensitivity C-reactive protein targets more frequent with the addition of ezetimibe to simvastatin and associated with better outcomes in IMPROVE-IT.

    Circulation · 2015 · 251 citations · Europe PMC · via Simvastatin

  4. New publication2015-06-03
    Ezetimibe Added to Statin Therapy after Acute Coronary Syndromes.

    The New England journal of medicine · 2015 · 3,026 citations · Europe PMC · via Simvastatin

  5. Safety communication2014-12-11

    Drug Safety Update: Simvastatin: dose limitations with concomitant amlodipine or diltiazem

    mhra · safety · mhra · via Simvastatin

  6. Safety communication2014-12-11

    Drug Safety Update: Simvastatin: increased risk of myopathy at high dose (80 mg)

    mhra · safety · mhra · via Simvastatin

  7. Safety communication2014-12-11

    Drug Safety Update: Simvastatin: updated advice on drug interactions

    mhra · safety · mhra · via Simvastatin

  8. New publication2014-03-19
    Effect of high-dose simvastatin on brain atrophy and disability in secondary progressive multiple sclerosis (MS-STAT): a randomised, placebo-controlled, phase 2 trial.

    Lancet (London, England) · 2014 · 322 citations · Europe PMC · via Simvastatin

  9. New publication2010-05-01
    Simvastatin as a treatment for pulmonary hypertension trial.

    American journal of respiratory and critical care medicine · 2010 · 96 citations · Europe PMC · via Simvastatin

  10. Label change2008-10-31

    Label change: SIMVASTATIN (ANDA076685)

    fda · regulatory · fda · via Simvastatin

  11. Label change2008-06-18

    Label change: SIMVASTATIN (ANDA076685)

    fda · regulatory · fda · via Simvastatin

  12. Regulatory approval2006-12-20

    Approval: SIMVASTATIN (ANDA076685)

    fda · regulatory · fda · via Simvastatin

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.