Elexacaftor

Summary

Elexacaftor (CHEMBL4298128) is an approved small molecule targeting CFTR; indicated across 1 condition including cystic fibrosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• Targets: 1 (CFTR)
• Indications: 1 condition
• Clinical trials: 7
• Chemistry: 597.7 Da · C26H34F3N7O4S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4298128
Name	Elexacaftor
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	134587348
Molecular formula	C26H34F3N7O4S
Molecular weight	597.7
InChIKey	MVRHVFSOIWFBTE-INIZCTEOSA-N

SMILES: C[C@H]1CC(N(C1)C2=C(C=CC(=N2)N3C=CC(=N3)OCC(C)(C)C(F)(F)F)C(=O)NS(=O)(=O)C4=CN(N=C4C)C)(C)C

IUPAC name: N-(1,3-dimethylpyrazol-4-yl)sulfonyl-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide

Also known as: Elexacaftor, Elexacaftor component of trikafta, Vx-445, VX-445, ELEXACAFTOR

Patent coverage: 243 distinct patent families (547 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 484 (88%) 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
CFTR	CFTR		7.15	0.1%	P13569

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Cystic fibrosis transmembrane conductance regulator.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 1 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CFTR	7.16	EC50	70	nM	CHEMBL_ACT_29226454

Target pathways

Aggregated over 1 target gene(s): CFTR.

Top Reactome pathways

11 total, by targets touching each:

Pathway	Targets	Genes
ABC-family protein mediated transport	1	CFTR
RHO GTPases regulate CFTR trafficking	1	CFTR
Defective CFTR causes cystic fibrosis	1	CFTR
Ub-specific processing proteases	1	CFTR
Cargo recognition for clathrin-mediated endocytosis	1	CFTR
Clathrin-mediated endocytosis	1	CFTR
RHOQ GTPase cycle	1	CFTR
Chaperone Mediated Autophagy	1	CFTR
Late endosomal microautophagy	1	CFTR
Aggrephagy	1	CFTR
Developmental Lineage of Pancreatic Ductal Cells	1	CFTR

Dominant GO biological processes

GO term	Targets
cholesterol biosynthetic process	1
water transport	1
bicarbonate transport	1
cholesterol transport	1
response to endoplasmic reticulum stress	1
transepithelial water transport	1
sperm capacitation	1
multicellular organismal-level water homeostasis	1
intracellular pH elevation	1
establishment of localization in cell	1
transmembrane transport	1
membrane hyperpolarization	1
positive regulation of enamel mineralization	1
cellular response to cAMP	1
amelogenesis	1

Indications & clinical

Indications

1 indication (0 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
cystic fibrosis	3	MONDO:0009061	MONDO:0009061

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
Not specified	3
PHASE3	2
PHASE4	1
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07148739	PHASE4	RECRUITING	Ensuring Access to Optimal Therapy in CF: The ENACT Study
NCT03525444	PHASE3	COMPLETED	A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03525548	PHASE3	COMPLETED	A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
NCT03227471	PHASE1/PHASE2	COMPLETED	A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis
NCT07363304	Not specified	RECRUITING	Impact of Elexacaftor-Tezacaftor-Ivacaftor Treatment on Metabolic, Epigenetic and Fecal Microbiota Profiles in People With Cystic Fibrosis.
NCT05576324	Not specified	UNKNOWN	Effect of the CFTR-modulating Triple Therapy Elexacaftor - Tezacaftor - Ivacaftor
NCT05699148	Not specified	UNKNOWN	Gut Imaging for Function and Transit in Cystic Fibrosis 3 Junior

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 8 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

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.

14 molecules share ≥1 primary target. Top 14 by shared-target count:

Molecule	Source	Status	Shared targets
IVACAFTOR	ChEMBL + PubChem	Phase 4 (approved)	CFTR
GLYBURIDE	ChEMBL	Phase 4 (approved)	CFTR
LUMACAFTOR	ChEMBL	Phase 4 (approved)	CFTR
TEZACAFTOR	ChEMBL	Phase 4 (approved)	CFTR
BAMOCAFTOR	ChEMBL	Phase 3	CFTR
QUERCETIN	ChEMBL	Phase 3	CFTR
RUTIN	ChEMBL	Phase 3	CFTR
GALICAFTOR	ChEMBL	Phase 2	CFTR
GENISTEIN	ChEMBL	Phase 2	CFTR
GLPG-2737	ChEMBL	Phase 2	CFTR
ICENTICAFTOR	ChEMBL	Phase 2	CFTR
NAVOCAFTOR	ChEMBL	Phase 2	CFTR
RISELCAFTOR	ChEMBL	Phase 2	CFTR
Tadalafil	PubChem	Approved	CFTR