Elagolix

Summary

Elagolix (CHEMBL1208155) is an approved small molecule (ATC H01CC03) targeting GNRHR; indicated across 2 conditions including endometriosis and polycystic ovary syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: H01CC03
• Targets: 1 (GNRHR)
• Indications: 2 conditions
• Clinical trials: 32
• Chemistry: 631.6 Da · C32H30F5N3O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1208155
Name	Elagolix
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	11250647
ATC	H01CC03
Molecular formula	C32H30F5N3O5
Molecular weight	631.6
InChIKey	HEAUOKZIVMZVQL-VWLOTQADSA-N

SMILES: CC1=C(C(=O)N(C(=O)N1CC2=C(C=CC=C2F)C(F)(F)F)C[C@@H](C3=CC=CC=C3)NCCCC(=O)O)C4=C(C(=CC=C4)OC)F

IUPAC name: 4-[[(1R)-2-[5-(2-fluoro-3-methoxyphenyl)-3-[[2-fluoro-6-(trifluoromethyl)phenyl]methyl]-4-methyl-2,6-dioxopyrimidin-1-yl]-1-phenylethyl]amino]butanoic acid

Also known as: Elagolix, NBI-56418, ELAGOLIX

Parent form; salt/anhydrous children: CHEMBL502182

Patent coverage: 373 distinct patent families (879 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 706 (80%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

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
GNRHR	GnRH1 receptor	Antagonist	9.05	0.1%	P30968

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Gonadotropin-releasing hormone receptor.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 1 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
GNRHR	9.22	EC50	0.6	nM	CHEMBL_ACT_29237209

Target pathways

Aggregated over 1 target gene(s): GNRHR.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
Hormone ligand-binding receptors	1	GNRHR
G alpha (q) signalling events	1	GNRHR

Dominant GO biological processes

GO term	Targets
G protein-coupled receptor signaling pathway	1
gonadotropin secretion	1
cellular response to hormone stimulus	1
signal transduction	1
cellular response to gonadotropin-releasing hormone	1

Indications & clinical

Indications

2 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
endometriosis	3	MONDO:0005133	EFO:0001065
polycystic ovary syndrome	2	MONDO:0008487	EFO:0000660

Clinical trials

Total trials: 32.

Phase distribution

Phase	Trials
PHASE3	15
PHASE2	9
PHASE4	4
EARLY_PHASE1	2
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06798571	PHASE4	RECRUITING	Role of Menopause in Thermoregulation
NCT07532876	PHASE4	ENROLLING_BY_INVITATION	Comparison of Elagolix and OCPs in Reducing Endometriosis Associated Pelvic Pain
NCT03886220	PHASE4	COMPLETED	A Study to Evaluate the Safety and Efficacy of Elagolix for the Management of Heavy Menstrual Bleeding Associated With Uterine Fibroids in Premenopausal Women
NCT05038878	PHASE4	TERMINATED	An Oral GnRH Antagonist to Treat Mild Autonomous Cortisol Excess (MACE) Due to Adrenal Adenomas in Postmenopausal Women
NCT04333576	PHASE3	ACTIVE_NOT_RECRUITING	Study Of Oral Elagolix Tablets In Combination With Combined Oral Contraceptive Capsules/Tablets To Assess Dysmenorrhea Response In Adult Female Participants With Endometriosis And Associated Moderate To Severe Pain
NCT06375811	PHASE3	RECRUITING	Pre-IVF Treatment With a GnRH Antagonist in Women With endometriosis_temp
NCT01620528	PHASE3	COMPLETED	A Clinical Study to Evaluate the Safety and Efficacy of Elagolix in Subjects With Moderate to Severe Endometriosis-Associated Pain
NCT01760954	PHASE3	COMPLETED	Study to Evaluate the Long-Term Safety and Efficacy of Elagolix in Adults With Moderate to Severe Endometriosis-Associated Pain
NCT01931670	PHASE3	COMPLETED	A Global Phase 3 Study to Evaluate the Safety and Efficacy of Elagolix in Subjects With Moderate to Severe Endometriosis-Associated Pain
NCT02143713	PHASE3	COMPLETED	Global Study to Evaluate the Long-Term Safety and Efficacy of Elagolix in Women With Moderate to Severe Endometriosis-associated Pain
NCT02654054	PHASE3	COMPLETED	Efficacy and Safety of Elagolix in Combination With Estradiol/Norethindrone Acetate for the Management of Heavy Menstrual Bleeding Associated With Uterine Fibroids in Premenopausal Women
NCT02691494	PHASE3	COMPLETED	Efficacy and Safety of Elagolix in Combination With Estradiol/Norethindrone Acetate for the Management of Heavy Menstrual Bleeding Associated With Uterine Fibroids in Premenopausal Women (Replicate Study)
NCT02925494	PHASE3	COMPLETED	An Extension Study to Evaluate the Efficacy and Safety of Elagolix in Premenopausal Women With Heavy Menstrual Bleeding Associated With Uterine Fibroids
NCT03213457	PHASE3	COMPLETED	A Clinical Study to Evaluate the Safety and Efficacy of Elagolix in Participants With Moderate to Severe Endometriosis-Associated Pain
NCT03271489	PHASE3	COMPLETED	Long-Term Safety Study of Elagolix in Combination With Estradiol/Norethindrone Acetate for the Management of Heavy Menstrual Bleeding Associated With Uterine Fibroids in Premenopausal Women
NCT03343067	PHASE3	TERMINATED	A Study to Evaluate Safety and Efficacy of Elagolix in Participants With Endometriosis With Associated Moderate to Severe Pain
NCT04173169	PHASE3	COMPLETED	Pre-IVF Treatment With a GnRH Antagonist in Women With Endometriosis
NCT05648669	PHASE3	UNKNOWN	A Study to Evaluate Safety and Efficacy of Elagolix in Patients With Moderate to Severe Endometriosis-Associated Pain
NCT06076486	PHASE3	COMPLETED	A Clinical Trial to Evaluate Efficacy and Safety of Elagolix Tablets in Women With Moderate or Severe Endometriosis-associated Pain
NCT00437658	PHASE2	COMPLETED	Elagolix Versus Subcutaneous Depot Medroxyprogesterone Acetate for the Treatment of Endometriosis
NCT00619866	PHASE2	COMPLETED	An Efficacy and Safety Study of Elagolix (NBI-56418) in Women With Endometriosis
NCT00797225	PHASE2	COMPLETED	Efficacy and Safety Study of Elagolix Versus Placebo or Leuprorelin Acetate in Endometriosis
NCT00973973	PHASE2	COMPLETED	Efficacy and Safety Study of Elagolix in Women With Endometriosis
NCT01441635	PHASE2	COMPLETED	Safety and Efficacy of Elagolix in Pre-Menopausal Women With Heavy Uterine Bleeding and Uterine Fibroids
NCT01817530	PHASE2	COMPLETED	Safety and Efficacy in Premenopausal Women With Heavy Menstrual Bleeding (HMB) Associated With Uterine Fibroids (UF)
NCT03951077	PHASE2	COMPLETED	Study of the Safety and Efficacy of Elagolix in Women With Polycystic Ovary Syndrome
NCT04039204	PHASE2	COMPLETED	Elagolix for Fertility Enhancement Clinical Trial
NCT04614246	PHASE2	TERMINATED	Study to Gather Information How Well Three Different Doses of BAY1817080 Given Twice Daily Over 12 Weeks Work in Comparison to an Inactive Pill (Placebo) and Elagolix in Women Suffering From Pain Related to a Condition Where the Tissue That Usually Grows Inside the Womb Grows Outside of the Womb
NCT01403038	PHASE1	COMPLETED	An Open-label Study of the Effects of Elagolix in Adult Premenopausal Females
NCT03746535	EARLY_PHASE1	RECRUITING	Cardiovascular Disease Risk in Women With Endometriosis
NCT04445025	EARLY_PHASE1	COMPLETED	Comparison Elagolix vs Depot Leuprolide Prior to Frozen Embryo Transfers in Patients With Endometriosis
NCT04856306	Not specified	UNKNOWN	Myomectomy vs Uterine Artery Embolization vs GnRh Antagonist for AUB-L

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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.

13 molecules share ≥1 primary target. Top 13 by shared-target count:

Molecule	Source	Status	Shared targets
CETRORELIX	ChEMBL + PubChem	Phase 4 (approved)	GNRHR
DEGARELIX	ChEMBL + PubChem	Phase 4 (approved)	GNRHR
GANIRELIX	ChEMBL + PubChem	Phase 4 (approved)	GNRHR
RELUGOLIX	ChEMBL + PubChem	Phase 4 (approved)	GNRHR
ABARELIX	ChEMBL	Phase 4 (approved)	GNRHR
GONADORELIN	ChEMBL	Phase 4 (approved)	GNRHR
LEUPROLIDE	ChEMBL	Phase 4 (approved)	GNRHR
LINZAGOLIX	ChEMBL	Phase 4 (approved)	GNRHR
ACYLINE	ChEMBL	Phase 2	GNRHR
SUFUGOLIX	ChEMBL	Phase 2	GNRHR
Belzutifan	PubChem	Approved	GNRHR
Deslorelin	PubChem	Approved	GNRHR
Triptorelin	PubChem	Approved	GNRHR