Tox | Related Drugs | 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

Predicted pharmacokinetic/toxicological properties (PK-AZ):

Property	Description	Value
kinase-safety-class	EIF2AK3,PDK1,PDK2,PDK4	4
kinase-selectivity-class	AXL,EIF2AK3,PDK4,PIK3CD,PIK3CG	5
pfas-class	PFAS structural alert (1 = True, 0 = False)	0

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:

Source	Code	Description
ATC	R07AX30	RESPIRATORY SYSTEM OTHER RESPIRATORY SYSTEM PRODUCTS OTHER RESPIRATORY SYSTEM PRODUCTS Other respiratory system products
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
FDA MoA	N0000190118	Cytochrome P450 3A Inducers
FDA MoA	N0000191264	P-Glycoprotein Inducers
CHEBI has role	CHEBI:66902	CFTR potentiator
CHEBI has role	CHEBI:71031	orphan drug

Related Drugs by ATC class:

R07AX Other respiratory system products 6 drugs

deutivacaftor elexacaftor ivacaftor nitric oxide tezacaftor vanzacaftor

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)

Formulation strength	Trade name	Applicant	Application number	Approval date	Type	Dose form	Route	Patent number	Patent expiration date	Patent use
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	7973038	Nov. 8, 2026	METHOD OF TREATING CYSTIC FIBROSIS USING N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3-CARBOXAMIDE AND 3-(6-(1-2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL) CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIC ACID
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	8741933	Nov. 8, 2026	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING IVACAFTOR AND LUMACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	7973038	Nov. 8, 2026	METHOD OF TREATING CYSTIC FIBROSIS USING N-(5-HYDROXY-2,4-DITERT-BUTYL-PHENYL)-4-OXO-1H-QUINOLINE-3-CARBOXAMIDE AND 3-(6-(1-2,2-DIFLUOROBENZO[D][1,3]DIOXOL-5-YL) CYCLOPROPANECARBOXAMIDO)-3-METHYLPYRIDIN-2-YL)BENZOIC ACID
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	8741933	Nov. 8, 2026	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING IVACAFTOR AND LUMACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	8741933	Nov. 8, 2026	METHOD OF TREATING CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE
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
188MG/PACKET;150MG/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
188MG/PACKET;150MG/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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	7973038	Nov. 8, 2026	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	8741933	Nov. 8, 2026	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	9670163	Dec. 28, 2026	METHOD OF TREATING A PATIENT HAVING CYSTIC FIBROSIS USING IVACAFTOR AND LUMACAFTOR
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	9931334	Dec. 28, 2026	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT AGE 6 OR OLDER HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (LESS THAN ABOUT 30% CRYSTALLINE) IVACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	9670163	Dec. 28, 2026	METHOD OF TREATING A PATIENT HAVING CYSTIC FIBROSIS USING IVACAFTOR AND LUMACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	9931334	Dec. 28, 2026	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT AGE 6 OR OLDER HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (LESS THAN ABOUT 30% CRYSTALLINE) 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
188MG/PACKET;150MG/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
188MG/PACKET;150MG/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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	9670163	Dec. 28, 2026	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	9931334	Dec. 28, 2026	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 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;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	8324242	Aug. 5, 2027	METHOD OF TREATING A PATIENT HAVING CYSTIC FIBROSIS USING IVACAFTOR AND LUMACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	8324242	Aug. 5, 2027	METHOD OF TREATING A PATIENT HAVING CYSTIC FIBROSIS USING IVACAFTOR AND LUMACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	8324242	Aug. 5, 2027	METHOD OF TREATING CYSTIC FIBROSIS
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
188MG/PACKET;150MG/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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	8324242	Aug. 5, 2027	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR AND IVACAFTOR
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	10597384	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 6 YEARS AND OLDER 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;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	11052075	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 6 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING THE TABLET ACCORDING TO CLAIM 1 OF U.S. PATENT NO. 11,052,075, WHERE THE TABLET FURTHER COMPRISES IVACAFTOR
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	12065432	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AND FORM I LUM AS RECITED IN, E.G., CLAIM 1 OF US 12065432
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	9150552	Dec. 4, 2028	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING IVACAFTOR AND FORM I LUMACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	10076513	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 12 YEARS OR OLDER WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING THE TABLET COMPRISING LUMACAFTOR AS RECITED IN CLAIM 1, 19, OR 21 OF U.S. PATENT NO. 10,076,513 AND IVACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	10597384	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 6 YEARS AND OLDER 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;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	11052075	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 6 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING THE TABLET ACCORDING TO CLAIM 1 OF U.S. PATENT NO. 11,052,075, WHERE THE TABLET FURTHER COMPRISES IVACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	12065432	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AND FORM I LUM AS RECITED IN, E.G., CLAIM 1 OF US 12065432
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	9150552	Dec. 4, 2028	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING IVACAFTOR AND FORM I LUMACAFTOR
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	12065432	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 1 YEAR TO 5 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AND FORM I LUM AS RECITED IN, E.G., CLAIM 1 OF US 12065432
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
188MG/PACKET;150MG/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
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	12065432	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 1 YEAR TO 5 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AND FORM I LUM AS RECITED IN, E.G., CLAIM 1 OF US 12065432
188MG/PACKET;150MG/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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	10597384	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 YEARS WHO IS 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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	12065432	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 1 YEAR TO 5 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AND FORM I LUM AS RECITED IN, E.G., CLAIM 1 OF US 12065432
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	9150552	Dec. 4, 2028	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR FORM I AND IVACAFTOR
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	8846718	July 2, 2029	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING IVACAFTOR AND FORM I LUMACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	8846718	July 2, 2029	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING IVACAFTOR AND FORM I LUMACAFTOR
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	8846718	July 2, 2029	METHOD OF TREATING CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE
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
188MG/PACKET;150MG/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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	8846718	July 2, 2029	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING LUMACAFTOR FORM I AND IVACAFTOR
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	11564916	Aug. 13, 2029	TREATMENT OF CF IN A PATIENT AGE 6 YEARS AND OLDER WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING THE COMPOSITION RECITED IN CLAIM 1 OF US 11564916
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	12458635	Aug. 13, 2029	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AS RECITED IN, E.G., CLAIM 1 OF US 12458635, AND FORM I LUM
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	11564916	Aug. 13, 2029	TREATMENT OF CF IN A PATIENT AGE 6 YEARS AND OLDER WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING THE COMPOSITION RECITED IN CLAIM 1 OF US 11564916
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	12458635	Aug. 13, 2029	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AS RECITED IN, E.G., CLAIM 1 OF US 12458635, AND FORM I LUM
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	12458635	Aug. 13, 2029	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 1 YEAR TO 5 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AS RECITED IN, E.G., CLAIM 1 OF US 12458635, AND FORM I LUM
188MG/PACKET;150MG/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
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	12458635	Aug. 13, 2029	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 1 YEAR TO 5 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AS RECITED IN, E.G., CLAIM 1 OF US 12458635, AND FORM I LUM
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	12458635	Aug. 13, 2029	TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 1 YEAR TO 5 YEARS WHO ARE HOMOZYGOUS FOR THE F508DEL CFTR GENE MUTATION COMPRISING ADMINISTERING AN EFFECTIVE AMOUNT OF IVA AS RECITED IN, E.G., CLAIM 1 OF US 12458635, AND FORM I LUM
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	9192606	Sept. 29, 2029	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING A DOSAGE UNIT AS DEFINED IN CLAIM 1 OF U.S. PATENT NO. 9,192,606
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	9192606	Sept. 29, 2029	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING A DOSAGE UNIT AS DEFINED IN CLAIM 1 OF U.S. PATENT NO. 9,192,606
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
188MG/PACKET;150MG/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
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	9192606	Sept. 29, 2029	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 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
125MG;100MG	ORKAMBI	VERTEX PHARMS INC	N206038	Sept. 28, 2016	RX	TABLET	ORAL	8716338	Sept. 20, 2030	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING THE DOSAGE UNIT OF CLAIM 1 OF U.S. PATENT NO. 8,716,338
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	8716338	Sept. 20, 2030	METHOD OF TREATING CYSTIC FIBROSIS IN A PATIENT, THE PATIENT HAVING THE F508DEL MUTATION IN CFTR, USING THE DOSAGE UNIT OF CLAIM 1 OF U.S. PATENT NO. 8,716,338
125MG;200MG	ORKAMBI	VERTEX PHARMS INC	N206038	July 2, 2015	RX	TABLET	ORAL	8716338	Sept. 20, 2030	METHOD OF TREATING CYSTIC FIBROSIS IN PATIENTS WHO HAVE THE F508DEL MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE.
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	8716338	Sept. 20, 2030	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 AS RECITED IN CLAIM 1 OF US PATENT 8716338 AND IVACAFTOR
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	8716338	Sept. 20, 2030	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 AS RECITED IN CLAIM 1 OF US PATENT 8716338 AND IVACAFTOR
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	8716338	Sept. 20, 2030	TREATMENT OF CYSTIC FIBROSIS IN A PATIENT AGE 1-5 YEARS WHO IS HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE USING THE DOSAGE UNIT COMPRISING LUMACAFTOR AS RECITED IN CLAIM 1 OF US PATENT 8716338 AND IVACAFTOR

Orange Book exclusivity data (new drug applications)

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	Dec. 13, 2027	ADDITIONAL CLINICAL TRIAL INFORMATION ADDED TO PEDIATRIC USE SUBSECTION
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	Dec. 13, 2027	ADDITIONAL CLINICAL TRIAL INFORMATION ADDED TO PEDIATRIC USE SUBSECTION
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	Dec. 13, 2027	ADDITIONAL CLINICAL TRIAL INFORMATION ADDED TO PEDIATRIC USE SUBSECTION
125MG/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	June 13, 2028	PEDIATRIC EXCLUSIVITY
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	June 13, 2028	PEDIATRIC EXCLUSIVITY
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	June 13, 2028	PEDIATRIC EXCLUSIVITY
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
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, 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/PACKET;100MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	March 2, 2030	PEDIATRIC EXCLUSIVITY
188MG/PACKET;150MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Aug. 7, 2018	RX	GRANULE	ORAL	March 2, 2030	PEDIATRIC EXCLUSIVITY
94MG/PACKET;75MG/PACKET	ORKAMBI	VERTEX PHARMS INC	N211358	Sept. 2, 2022	RX	GRANULE	ORAL	March 2, 2030	PEDIATRIC EXCLUSIVITY

Bioactivity Summary:

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

External reference:

ID	Source
D10134	KEGG_DRUG
4034600	VUID
N0000191736	NUI
4034600	VANDF
CHEBI:90951	CHEBI
VX8	PDB_CHEM_ID
CHEMBL2103870	ChEMBL_ID
C569105	MESH_SUPPLEMENTAL_RECORD_UI
7481	IUPHAR_LIGAND_ID
DB09280	DRUGBANK_ID
1655922	RXNORM
31119	MMSL
d08371	MMSL
016231	NDDF
716073005	SNOMEDCT_US
C3852684	UMLSCUI
9449	INN_ID
16678941	PUBCHEM_CID
EGP8L81APK	UNII

Pharmaceutical products:

Product	Category	Ingredients	NDC	Form	Quantity	Route	Marketing	Label
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-122	GRANULE	75 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-122	GRANULE	75 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-500	GRANULE	150 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-500	GRANULE	150 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-700	TABLET, FILM COATED	100 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-700	TABLET, FILM COATED	100 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-809	TABLET, FILM COATED	200 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-809	TABLET, FILM COATED	200 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-900	GRANULE	100 mg	ORAL	NDA	33 sections
ORKAMBI	HUMAN PRESCRIPTION DRUG LABEL	2	51167-900	GRANULE	100 mg	ORAL	NDA	33 sections

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lumacaftor 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |

Description:

Drug dosage:

ADMET properties:

Predicted pharmacokinetic/toxicological properties (PK-AZ):

Approvals:

FDA Adverse Event Reporting System (Female)

FDA Adverse Event Reporting System (Male)

FDA Adverse Event Reporting System (Geriatric)

FDA Adverse Event Reporting System (Pediatric)

Pharmacologic Action:

Related Drugs by ATC class:

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

🐶 Veterinary Drug Use

🐶 Veterinary products

Acid dissociation constants calculated using MoKa v3.0.0

Orange Book patent data (new drug applications)

Orange Book exclusivity data (new drug applications)

Bioactivity Summary:

External reference:

Pharmaceutical products: