Alirocumab

Summary

Alirocumab (CHEMBL2109540) is an approved antibody (ATC C10AX14) targeting PCSK9; indicated across 11 conditions including familial hypercholesterolemia and cardiovascular disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Antibody
• ATC class: C10AX14
• Targets: 1 (PCSK9)
• Indications: 11 conditions
• Clinical trials: 89

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2109540
Name	Alirocumab
Type	Antibody
Max phase	4
ATC	C10AX14

Also known as: Alirocumab, Praluent, REGN-727, REGN727, SAR-236553, SAR236553, ALIROCUMAB

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	9.42	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

11 indications (6 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
atherosclerosis	4	MONDO:0005311	EFO:0003914
coronary artery disorder	4	MONDO:0005010	EFO:0001645
diabetes mellitus	3	MONDO:0005015	EFO:0000400
nephrotic syndrome	2	MONDO:0005377	EFO:0004255

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

Clinical trials

Total trials: 89.

Phase distribution

Phase	Trials
PHASE3	32
PHASE4	16
PHASE1	15
PHASE2	14
Not specified	11
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05292404	PHASE4	RECRUITING	Impact of Early PCSK9 Inhibitor Treatment on Heart After Acute Myocardium Infarction
NCT06083961	PHASE4	NOT_YET_RECRUITING	The Effect of Early Administration of PCSK9 Inhibitor to Acute Ischemic Stroke Patients Associated With Atherosclerosis on the Stroke Prognosis and Lipid Profile
NCT07581808	PHASE4	NOT_YET_RECRUITING	Dual PCSK9 Inhibition With Inclisiran and Alirocumab in Secondary Prevention
NCT02642159	PHASE4	COMPLETED	Efficacy and Safety of Alirocumab Versus Usual Care on Top of Maximally Tolerated Statin Therapy in Patients With Type 2 Diabetes and Mixed Dyslipidemia (ODYSSEY DM-Dyslipidemia)
NCT02938949	PHASE4	COMPLETED	Alirocumab in Patients With Acute Myocardial Infarction
NCT02957682	PHASE4	COMPLETED	Evaluating Effect of the Study Drug Praluent (Alirocumab) on Neurocognitive Function When Compared to Placebo
NCT02959047	PHASE4	COMPLETED	A Trial of Alirocumab and Plaque Regression in Peripheral Arterial Disease
NCT02984982	PHASE4	COMPLETED	Evaluation of Effect of Alirocumab on Coronary Atheroma Volume in Japanese Patients Hospitalized for Acute Coronary Syndrome With Hypercholesterolemia
NCT02992301	PHASE4	UNKNOWN	Assessment of Atherosclerotic Plaque Characteristics Change by DCE-MRI With Alirocumab
NCT03529253	PHASE4	UNKNOWN	Effect of Alirocumab(Proprotein Convertase Subtilisin/Kexin type9 Inhibitor) and Rosuvastatin or Rosuvastatin Alone on Lipid Core Plaques in Coronary Artery Disease Evaluated by Near-infrared Spectroscopy Intravascular Ultrasound
NCT03542110	PHASE4	TERMINATED	The Alirocumab for Stopping Atherosclerosis Progression in Saphenous Vein Grafts (ASAP-SVG) Pilot Trial
NCT03552432	PHASE4	UNKNOWN	The Efficacy of Alirocumab for Thin-cap fIbroatheroma in Patients With Coronary Artery Disease Estimated by Optical Coherence Tomography
NCT03694197	PHASE4	TERMINATED	Long Term Safety Study of PRALUENT
NCT03750760	PHASE4	WITHDRAWN	Early Alirocumab to Reduce LDL-C in Myocardial Infarction
NCT04193306	PHASE4	UNKNOWN	Efficacy and Safety Of Alirocumab to Prevent Early Cardiac Allograft Vasculopathy in Recent Heart Transplant Recipients
NCT05465278	PHASE4	COMPLETED	Alirocumab and Plaque Burden In Familial Hypercholesterolaemia
NCT03480568	PHASE3	RECRUITING	Alirocumab in Patients on a Stable Dialysis Regimen
NCT07586540	PHASE3	NOT_YET_RECRUITING	Alirocumab for Stabilisation of Symptomatic Vulnerable Carotid Plaque
NCT01507831	PHASE3	COMPLETED	Long-term Safety and Tolerability of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in High Cardiovascular Risk Patients With Hypercholesterolemia (ODYSSEY Long Term)
NCT01617655	PHASE3	COMPLETED	Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia (ODYSSEY HIGH FH)
NCT01623115	PHASE3	COMPLETED	Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy
NCT01644175	PHASE3	COMPLETED	Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With High Cardiovascular Risk and Hypercholesterolemia (ODYSSEY COMBO I)
NCT01644188	PHASE3	COMPLETED	Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Ezetimibe on Top of Statin in High Cardiovascular Risk Patients With Hypercholesterolemia (ODYSSEY COMBO II)
NCT01644474	PHASE3	COMPLETED	Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Ezetimibe in Patients With Hypercholesterolemia
NCT01663402	PHASE3	COMPLETED	ODYSSEY Outcomes: Evaluation of Cardiovascular Outcomes After an Acute Coronary Syndrome During Treatment With Alirocumab
NCT01709500	PHASE3	COMPLETED	Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy)
NCT01709513	PHASE3	COMPLETED	Study of Alirocumab (REGN727/SAR236553) in Patients With Primary Hypercholesterolemia and Moderate, High, or Very High Cardiovascular (CV) Risk, Who Are Intolerant to Statins (ODYSSEY ALTERNATIVE)
NCT01730040	PHASE3	COMPLETED	Study of the Efficacy and Safety of Alirocumab (REGN727/SAR236553) in Combination With Other Lipid-modifying Treatment (LMT) (ODYSSEY OPTIONS I)
NCT01730053	PHASE3	COMPLETED	Study of Alirocumab (REGN727/SAR236553) added-on to Rosuvastatin Versus Other Lipid Modifying Treatments (LMT) (ODYSSEY OPTIONS II)
NCT01926782	PHASE3	COMPLETED	Study to Evaluate the Efficacy and Safety of an Every Four Weeks Treatment Regimen of Alirocumab (REGN727/ SAR236553) in Patients With Primary Hypercholesterolemia (ODYSSEY CHOICE 1)
NCT01954394	PHASE3	COMPLETED	Open Label Study of Long Term Safety Evaluation of Alirocumab
NCT02023879	PHASE3	COMPLETED	Phase III Study To Evaluate Alirocumab in Patients With Hypercholesterolemia Not Treated With a Statin (ODYSSEY CHOICE II)
NCT02107898	PHASE3	COMPLETED	Efficacy and Safety Evaluation of Alirocumab in Patients With Heterozygous Familial Hypercholesterolemia or High Cardiovascular Risk Patients With Hypercholesterolemia on Lipid Modifying Therapy (ODYSSEY JAPAN)
NCT02289963	PHASE3	COMPLETED	Evaluation of Alirocumab in Addition to Lipid-Modifying Therapy in Patients With High Cardiovascular Risk and Hypercholesterolemia in South Korea and Taiwan
NCT02326220	PHASE3	COMPLETED	Study of Alirocumab (REGN727/SAR236553) in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) Undergoing Low-density Lipoprotein (LDL) Apheresis Therapy
NCT02476006	PHASE3	COMPLETED	Safety, Tolerability, and Effect of Alirocumab in High Cardiovascular Risk Patients With Severe Hypercholesterolemia Not Adequately Controlled With Conventional Lipid-modifying Therapies (ODYSSEY APPRISE)
NCT02584504	PHASE3	COMPLETED	Efficacy and Safety of Alirocumab in Patients With Hypercholesterolemia Not Adequately Controlled With Non-statin Lipid Modifying Therapy or the Lowest Strength of Statin
NCT02585778	PHASE3	COMPLETED	Efficacy and Safety of Alirocumab Versus Placebo on Top of Maximally Tolerated Lipid Lowering Therapy in Patients With Hypercholesterolemia Who Have Type 1 or Type 2 Diabetes and Are Treated With Insulin (ODYSSEY DM - Insulin)
NCT02715726	PHASE3	COMPLETED	Evaluation of Alirocumab Versus Ezetimibe on Top of Statin in Asia in High Cardiovascular Risk Patients With Hypercholesterolemia
NCT03067844	PHASE3	COMPLETED	Vascular Effects of Alirocumab in Acute MI-Patients

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