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

Stem definitionDrug idCAS RN
cystic fibrosis transmembrane regulator (CFTR) protein modulators 5010 936727-05-8

Description:

MoleculeDescription

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:

DateAgencyCompanyOrphan
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:

SourceCodeDescription
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 cystic fibrosis transmembrane conductance regulator potentiators
CHEBI has role CHEBI:71031 orphan drugs

Drug Use (View source of the data)

DiseaseRelationSNOMED_IDDOID
Cystic fibrosis of the lung indication 86555001




🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation levelDissociation constantType (acidic/basic)
pKa1 4.67 acidic
pKa2 10.91 acidic
pKa3 4.06 Basic

Orange Book patent data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent 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
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
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
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 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 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 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 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;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
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
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

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
125MG;100MG ORKAMBI VERTEX PHARMS INC N206038 Sept. 28, 2016 RX TABLET ORAL July 2, 2022 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR F508DEL MUTATION IN THE CFTR GENE
125MG;200MG ORKAMBI VERTEX PHARMS INC N206038 July 2, 2015 RX TABLET ORAL July 2, 2022 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR 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
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
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

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
Cystic fibrosis transmembrane conductance regulator Ion channel PHARMACOLOGICAL CHAPERONE EC50 6.46 SCIENTIFIC LITERATURE DRUG LABEL

External reference:

IDSource
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
9449 INN_ID
DB09280 DRUGBANK_ID
EGP8L81APK UNII
16678941 PUBCHEM_CID
1655922 RXNORM
31119 MMSL
d08371 MMSL
716073005 SNOMEDCT_US
C3852684 UMLSCUI
016231 NDDF

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
ORKAMBI HUMAN PRESCRIPTION DRUG LABEL 2 51167-500 GRANULE 150 mg ORAL NDA 30 sections
ORKAMBI HUMAN PRESCRIPTION DRUG LABEL 2 51167-700 TABLET, FILM COATED 100 mg ORAL NDA 30 sections
ORKAMBI HUMAN PRESCRIPTION DRUG LABEL 2 51167-809 TABLET, FILM COATED 200 mg ORAL NDA 30 sections
ORKAMBI HUMAN PRESCRIPTION DRUG LABEL 2 51167-900 GRANULE 100 mg ORAL NDA 30 sections