elexacaftor 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definition	Drug id	CAS RN
cystic fibrosis transmembrane regulator (CFTR) protein modulators, correctors, and amplifiers	5372	2216712-66-0

Description:

Molecule	Description
Molfile Inchi Smiles Synonyms: elexacaftor	Elexacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of F508del-CFTR to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Ivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface. The combined effect of elexacaftor, tezacaftor and ivacaftor is increased quantity and function of F508del-CFTR at the cell surface, resulting in increased CFTR activity as measured by CFTR mediated chloride transport. Molecular weight: 597.66 Formula: C26H34F3N7O4S CLOGP: 5.26 LIPINSKI: 2 HAC: 11 HDO: 1 TPSA: 124.24 ALOGS: -4.49 ROTB: 8 Status: ONP Legend: OFP - off patent OFM - off market ONP - on patent

Molecule

Description

Molfile Inchi Smiles

Synonyms:

elexacaftor

Elexacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of F508del-CFTR to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Ivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface. The combined effect of elexacaftor, tezacaftor and ivacaftor is increased quantity and function of F508del-CFTR at the cell surface, resulting in increased CFTR activity as measured by CFTR mediated chloride transport.

Molecular weight: 597.66
Formula: C26H34F3N7O4S
CLOGP: 5.26
LIPINSKI: 2
HAC: 11
HDO: 1
TPSA: 124.24
ALOGS: -4.49
ROTB: 8

Status: ONP

Legend:
OFP - off patent
OFM - off market
ONP - on patent

Drug dosage:

None

ADMET properties:

Property	Value	Reference
BDDCS (Biopharmaceutical Drug Disposition Classification System)	1	Bocci G, Oprea TI, Benet LZ

Approvals:

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Date	Agency	Company	Orphan
Aug. 21, 2020	EMA	Vertex Pharmaceuticals (Ireland) Limited
Oct. 10, 2019	FDA	VERTEX PHARMS INC

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FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

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Source	Code	Description
ATC	R07AX32	RESPIRATORY SYSTEM OTHER RESPIRATORY SYSTEM PRODUCTS OTHER RESPIRATORY SYSTEM PRODUCTS Other respiratory system products
MeSH PA	D065101	Chloride Channel Agonists
MeSH PA	D049990	Membrane Transport Modulators

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Drug Use | Suggest Off label Use Form| |View source of the data|

Disease	Relation	SNOMED_ID	DOID
Cystic fibrosis	indication	190905008	DOID:1485

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation level	Dissociation constant	Type (acidic/basic)
pKa1	4.64	acidic
pKa2	1.88	Basic

Orange Book patent data (new drug applications)

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Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Patent number	Patent expiration date	Patent use
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	8629162	June 24, 2025	TREATMENT OF A MODERATE TO MILD CLINICAL PHENOTYPE OF CF IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CFTR GENE OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA WITH ELX, TEZ, AND IVA
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	8354427	July 6, 2026	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE A R117H MUTATION IN THE CFTR GENE WITH ELEXACAFTOR, TEZACAFTOR, AND IVACAFTOR
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	9670163	Dec. 28, 2026	TREATMENT OF CF IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA USING A SOLID COMPOSITION COMPRISING ELX, TEZ, AMORPHOUS IVA, AND < ~30% CRYSTALLINE IVA
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	9931334	Dec. 28, 2026	TREATMENT OF CF IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA USING A SOLID COMPOSITION COMPRISING ELX, TEZ, AMORPHOUS IVA, AND < ~30% CRYSTALLINE IVA
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	10022352	April 9, 2027	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CFTR GENE WITH AN EFFECTIVE AMOUNT OF A PHARMACEUTICAL COMPOSITION COMPRISING ELEXACAFTOR, TEZACAFTOR, AND IVACAFTOR
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	10239867	April 9, 2027	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CFTR GENE OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA WITH AN EFFECTIVE AMOUNT OF ELX, TEZ, AND IVA
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	11639347	April 9, 2027	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE IN THE CFTR GENE AT LEAST ONE F508DEL MUTATION OR A RESPONSIVE MUTATION BASED ON IN VITRO DATA WITH AN EFFECTIVE AMOUNT OF ELX, TEZ, AND IVA
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	9974781	April 9, 2027	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CFTR GENE OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA WITH ELEXACAFTOR, TEZACAFTOR, AND IVACAFTOR
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	8598181	May 1, 2027	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CFTR GENE OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA WITH ELEXACAFTOR, TEZACAFTOR, AND IVACAFTOR
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	8324242	Aug. 5, 2027	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 2 TO <6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CFTR GENE OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA WITH ELEXACAFTOR, TEZACAFTOR, AND IVACAFTOR

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Orange Book exclusivity data (new drug applications)

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Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Exclusivity date	Description
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	Oct. 21, 2024	NEW CHEMICAL ENTITY
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	April 26, 2026	NEW PRODUCT
100MG, 75MG, 50MG;75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N217660	April 26, 2023	RX	GRANULES	ORAL	April 26, 2030	TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 2 YEARS TO LESS THAN 6 YEARS WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA
100MG,75MG,50MG; 150MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N212273	Oct. 21, 2019	RX	TABLET	ORAL	Feb. 12, 2023	NEW CHEMICAL ENTITY
100MG,75MG,50MG; 150MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N212273	Oct. 21, 2019	RX	TABLET	ORAL	June 8, 2024	NEW PATIENT POPULATION
100MG,75MG,50MG; 150MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N212273	Oct. 21, 2019	RX	TABLET	ORAL	Oct. 21, 2024	NEW CHEMICAL ENTITY
100MG,75MG,50MG; 150MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N212273	Oct. 21, 2019	RX	TABLET	ORAL	Oct. 21, 2026	INDICATED FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 12 YEARS AND OLDER WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE
100MG,75MG,50MG; 150MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N212273	Oct. 21, 2019	RX	TABLET	ORAL	Dec. 21, 2027	TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 12 YEARS AND OLDER WHO HAVE A MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA
100MG,75MG,50MG; 150MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N212273	Oct. 21, 2019	RX	TABLET	ORAL	June 8, 2028	FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 6 THROUGH 11 YEARS OLD WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE OR A MUTATION IN THE CFTR GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA
50MG,37.5MG,25MG; 75MG	TRIKAFTA (COPACKAGED)	VERTEX PHARMS INC	N212273	June 8, 2021	RX	TABLET	ORAL	Feb. 12, 2023	NEW CHEMICAL ENTITY

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Bioactivity Summary:

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Target	Class	Pharos	UniProt	Action	Type	Activity value (-log[M])	Mechanism action	Bioact source	MoA source
Cystic fibrosis transmembrane conductance regulator	Ion channel	P13569	CFTR_HUMAN		EC50	7.15		IUPHAR	DRUG LABEL

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External reference:

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ID	Source
WJX	PDB_CHEM_ID
018170	NDDF
10552	IUPHAR_LIGAND_ID
11180	INN_ID
134587348	PUBCHEM_CID
2256951	RXNORM
37631	MMSL
4038884	VANDF
827070004	SNOMEDCT_US
C000629074	MESH_SUPPLEMENTAL_RECORD_UI

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Pharmaceutical products:

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