Evolocumab

Summary

Evolocumab (CHEMBL2364655) is an approved antibody (ATC C10AX13) targeting PCSK9; indicated across 21 conditions including familial hypercholesterolemia and cardiovascular disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Antibody
• ATC class: C10AX13
• Targets: 1 (PCSK9)
• Indications: 21 conditions
• Clinical trials: 114

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2364655
Name	Evolocumab
Type	Antibody
Max phase	4
ATC	C10AX13

Also known as: AMG-145, Evolocumab, Repatha, Repatha sureclick, EVOLOCUMAB

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
PCSK9	proprotein convertase subtilisin/kexin type 9	Binding	9.72	4.6%	Q8NBP7

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): PCSK9.

Top Reactome pathways

4 total, by targets touching each:

Pathway	Targets	Genes
Regulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs)	1	PCSK9
VLDLR internalisation and degradation	1	PCSK9
Post-translational protein phosphorylation	1	PCSK9
LDL clearance	1	PCSK9

Dominant GO biological processes

GO term	Targets
kidney development	1
liver development	1
negative regulation of receptor recycling	1
negative regulation of receptor internalization	1
positive regulation of receptor internalization	1
triglyceride metabolic process	1
phospholipid metabolic process	1
apoptotic process	1
lysosomal transport	1
cholesterol metabolic process	1
cellular response to starvation	1
negative regulation of low-density lipoprotein particle clearance	1
protein autoprocessing	1
neurogenesis	1
neuron differentiation	1

Indications & clinical

Indications

21 indications (8 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
familial hypercholesterolemia	4	MONDO:0005439	EFO:0004911
cardiovascular disorder	4	MONDO:0004995	EFO:0000319
coronary artery disorder	4	MONDO:0005010	EFO:0001645
myocardial infarction	4	MONDO:0005068	EFO:0000612
stroke disorder	4	MONDO:0005098	EFO:0000712
acute coronary syndrome	3	MONDO:0005542	EFO:0005672
severe acute respiratory syndrome	3	MONDO:0005091	MONDO:0100096
hyperlipidemia	3	MONDO:0021187	MONDO:0021187
heart failure	2	MONDO:0005252	EFO:0003144
ST-elevation myocardial infarction	2	MONDO:0041656	EFO:0008585
lung neoplasm	2	MONDO:0021117	MONDO:0008903
renal cell adenocarcinoma	2	MONDO:0005549	EFO:0005708
non-small cell lung carcinoma	1	MONDO:0005233	EFO:0003060
glioblastoma	0	MONDO:0018177	EFO:0000519
abdominal aortic aneurysm	0	MONDO:0005350	EFO:0004214

6 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 114.

Phase distribution

Phase	Trials
PHASE3	35
PHASE4	32
PHASE2	21
Not specified	16
PHASE1	3
EARLY_PHASE1	3
PHASE2/PHASE3	2
PHASE1/PHASE2	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04951856	PHASE4	ACTIVE_NOT_RECRUITING	Evolocumab or Normal Strategies to Reach LDL Objectives in Acute Myocardial Infarction Upbound to PCI
NCT05152888	PHASE4	RECRUITING	The Impact of Pcsk-9 Inhibition on PET CFR in Patients at High CV Risk
NCT05284747	PHASE4	ACTIVE_NOT_RECRUITING	EVOLVE-MI: EVOLocumab Very Early After Myocardial Infarction
NCT05641753	PHASE4	RECRUITING	Cholesterol Lowering and Residual Risk in Diabetes, Type 1
NCT07612774	PHASE4	RECRUITING	CAPER-EVO: a Randomized Serial PCCT Trial of Early Evolocumab After ACS
NCT02948777	PHASE4	COMPLETED	Effects of PCSK9 Inhibition by Evolocumab on Postprandial Lipid Metabolism in Type 2 Diabetes
NCT03096288	PHASE4	COMPLETED	Impact of Evolocumab on the Effects of Clopidogrel in Patients With High On-Treatment Platelet Reactivity
NCT03258281	PHASE4	TERMINATED	Effects of Evolocumab on Platelet Reactivity in Patients With Diabetes Mellitus
NCT03331666	PHASE4	TERMINATED	Impact of LDL-cholesterol Lowering on Platelet Activation
NCT03403374	PHASE4	COMPLETED	Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia
NCT03829046	PHASE4	COMPLETED	The Effects of Evolocumab in Patients With Diabetes and Atherosclerotic Vascular Disease
NCT03851263	PHASE4	WITHDRAWN	Multislice Computed Tomography Assessment of PCSK9 Inhibition on Coronary Perfusion
NCT03900026	PHASE4	COMPLETED	Effect of Evolocumab on Saphenous Vein Graft Patency Following Coronary Artery Bypass Surgery
NCT03932721	PHASE4	COMPLETED	EXpanded Combination of Evolocumab Plus Empagliflozin on Diabetes: EXCEED-BHS3 Trial
NCT04073134	PHASE4	TERMINATED	The CHORAL Flow Study
NCT04100434	PHASE4	UNKNOWN	Effect of Evolocumab Added to Moderate-Intensity Statin Therapy on LDL-C Lowering and Cardiovascular Adverse Events in Patients With Acute Coronary Syndrome
NCT04397653	PHASE4	UNKNOWN	Evolocumab Plus Ezetimibe in High Risk Haemodialized Statin Intolerant Patients
NCT04510844	PHASE4	WITHDRAWN	Evolocumab In Advanced Chronic Kidney Disease Trial
NCT04539223	PHASE4	UNKNOWN	A Study of Evolocumab on Carotid Artery Atherosclerotic Plaque Morphology Prior to Carotid EndArterectomy
NCT04573777	PHASE4	TERMINATED	Reducing Intracranial atheroSclErosis With Repatha
NCT04608474	PHASE4	COMPLETED	Lipid Management in Renal Transplant Recipients Using Evolocumab.
NCT04659525	PHASE4	UNKNOWN	Evolocumab Plus Ezetimibe in Haemodialized Statin-intolerant Patients With Hypercholesterolemia
NCT04710368	PHASE4	COMPLETED	Effect of Evolocumab on Coronary Plaque Characteristics
NCT04719221	PHASE4	UNKNOWN	Impact of Evolocumab as an Additional Lipid-lowering Therapy to Changes in Lipid Core Burden Index of Non-culprit Vulnerable Plaque in Patients Who Underwent Percutaneous Coronary Intervention for the Acute Coronary Syndrome
NCT04730973	PHASE4	UNKNOWN	CARotid plaqUe StabilizatiOn and Regression With Evolocumab.
NCT05579418	PHASE4	UNKNOWN	The Safety and Long-Term Clinical Benefit of PCSK9i in STEMI Patients
NCT05585151	PHASE4	UNKNOWN	High-Resolution Assessment of Extracranial Plaques in a Multiple Centers Evolocumab Randomized Study
NCT05613426	PHASE4	TERMINATED	Effect of Very Early and Rapid Lowering Cholesterol With Evolocumab on Left Ventricular Remodeling in Patients With Anterior STEMI Undergoing Primary PCI
NCT05661552	PHASE4	COMPLETED	Effect of Early Initiation of Evolocumab on Lipid Profiles Changes in Patients With ACS Undergoing PCI
NCT05697185	PHASE4	UNKNOWN	Safety and Efficacy of Evolocumab in Ischemic Stroke
NCT05976893	PHASE4	UNKNOWN	Study on the Composite Endpoint Event of PCSK9 Inhibitor in Patients With Very High Risk of ASCVD and Cancer
NCT06231459	PHASE4	COMPLETED	Expression of Pro- and Anti-inflammatory Cytokines During Anti-PCSK9 in Familial Hypercholesterolemia
NCT01516879	PHASE3	COMPLETED	Durable Effect of PCSK9 Antibody CompARed wiTh placEbo Study
NCT01588496	PHASE2/PHASE3	COMPLETED	Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities
NCT01624142	PHASE2/PHASE3	COMPLETED	Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders
NCT01763827	PHASE3	COMPLETED	Monoclonal Antibody Against PCSK9 to Reduce Elevated LDL-C in Subjects Currently Not Receiving Drug Therapy for Easing Lipid Levels-2
NCT01763866	PHASE3	COMPLETED	LDL-C Assessment With PCSK9 Monoclonal Antibody Inhibition Combined With Statin Therapy-2
NCT01763905	PHASE3	COMPLETED	Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects -2
NCT01763918	PHASE3	COMPLETED	Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2
NCT01764633	PHASE3	COMPLETED	Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

1 molecules share ≥1 primary target. Top 1 by shared-target count:

Molecule	Source	Status	Shared targets
NILOTINIB	ChEMBL + PubChem	Phase 4 (approved)	PCSK9