lumacaftor 🐶 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	5010	936727-05-8

Description:

Molecule	Description
Molfile Inchi Smiles Synonyms: lumacaftor VX-809 VRT-826809	Lumacaftor improves the conformational stability of F508del-CFTR, resulting in increased processing and trafficking of mature protein to the cell surface. Molecular weight: 452.41 Formula: C24H18F2N2O5 CLOGP: 5.15 LIPINSKI: 1 HAC: 7 HDO: 2 TPSA: 97.75 ALOGS: -5.08 ROTB: 5 Status: ONP Legend: OFP - off patent OFM - off market ONP - on patent

Molecule

Description

Molfile Inchi Smiles

Synonyms:

lumacaftor
VX-809
VRT-826809

Lumacaftor improves the conformational stability of F508del-CFTR, resulting in increased processing and trafficking of mature protein to the cell surface.

Molecular weight: 452.41
Formula: C24H18F2N2O5
CLOGP: 5.15
LIPINSKI: 1
HAC: 7
HDO: 2
TPSA: 97.75
ALOGS: -5.08
ROTB: 5

Status: ONP

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

Drug dosage:

None

ADMET properties:

None

Approvals:

Date	Agency	Company	Orphan
July 2, 2015	FDA	VERTEX PHARMS INC

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	R07AX30	RESPIRATORY SYSTEM OTHER RESPIRATORY SYSTEM PRODUCTS OTHER RESPIRATORY SYSTEM PRODUCTS Other respiratory system products
CHEBI has role	CHEBI:66902	cystic fibrosis transmembrane conductance regulator potentiators
CHEBI has role	CHEBI:71031	orphan drugs
FDA MoA	N0000185503	P-Glycoprotein Inhibitors
FDA MoA	N0000185504	Cytochrome P450 2C9 Inhibitors
FDA MoA	N0000185507	Cytochrome P450 2C9 Inducers
FDA MoA	N0000185607	Cytochrome P450 2C19 Inducers
FDA MoA	N0000187062	Cytochrome P450 2C8 Inhibitors
FDA MoA	N0000187063	Cytochrome P450 2C8 Inducers
FDA MoA	N0000187064	Cytochrome P450 2B6 Inducers

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

Disease	Relation	SNOMED_ID	DOID
Cystic fibrosis of the lung	indication	86555001

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation level	Dissociation constant	Type (acidic/basic)
pKa1	4.67	acidic
pKa2	10.91	acidic
pKa3	4.06	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
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	7973038	Nov. 8, 2026	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	8741933	Nov. 8, 2026	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	9670163	Dec. 28, 2026	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS AND LESS THAN ABOUT 30% CRYSTALLINE IVACAFTOR
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	9931334	Dec. 28, 2026	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS AND LESS THAN ABOUT 30% CRYSTALLINE IVACAFTOR
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	8324242	Aug. 5, 2027	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	10597384	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 2 TO 5 YEARS OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING A PHARMACEUTICAL COMPOSITION ACCORDING TO CLAIM 2 OF U.S. PATENT NO. 10,597,384, FURTHER COMPRISING IVACAFTOR
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	9150552	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR FORM I AND IVACAFTOR
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	8846718	July 2, 2029	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR FORM I AND IVACAFTOR
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	11564916	Aug. 13, 2029	TREATMENT OF CF IN A PATIENT AGE 1 TO <6 YEARS AND WEIGHING 7 KG OR MORE WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING THE COMPOSITION RECITED IN CLAIM 1 OF US 11564916
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	9192606	Sept. 29, 2029	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 2-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING THE DOSAGE UNIT COMPRISING LUMACAFTOR AND IVACAFTOR AS RECITED IN CLAIM 1 OF US PATENT 9192606

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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
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	Aug. 7, 2025	FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 2 THROUGH 5 YEARS OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	Sept. 2, 2025	NEW PATIENT POPULATION
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	Sept. 2, 2029	TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 1 YEAR TO LESS THAN 2 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	Sept. 28, 2023	TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6-11 YEAR OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	Sept. 28, 2023	TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6-11 YEAR OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	Aug. 7, 2025	FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 2 THROUGH 5 YEARS OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	Sept. 2, 2025	NEW PATIENT POPULATION
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	Sept. 2, 2029	TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 1 YEAR TO LESS THAN 2 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	Sept. 2, 2025	NEW STRENGTH
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	Sept. 2, 2029	TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 1 YEAR TO LESS THAN 2 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE

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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	PHARMACOLOGICAL CHAPERONE	EC50	6.46		SCIENTIFIC LITERATURE	DRUG LABEL

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External reference:

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ID	Source
VX8	PDB_CHEM_ID
016231	NDDF
1655922	RXNORM
16678941	PUBCHEM_CID
31119	MMSL
4034600	VUID
4034600	VANDF
716073005	SNOMEDCT_US
7481	IUPHAR_LIGAND_ID
9449	INN_ID

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Pharmaceutical products:

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Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-122	GRANULE	75 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-122	GRANULE	75 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-500	GRANULE	150 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-500	GRANULE	150 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-500	GRANULE	150 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-700	TABLET, FILM COATED	100 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-700	TABLET, FILM COATED	100 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-700	TABLET, FILM COATED	100 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-809	TABLET, FILM COATED	200 mg	ORAL	NDA	34 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-809	TABLET, FILM COATED	200 mg	ORAL	NDA	34 sections

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