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 |
|---|---|
|
Synonyms:
|
Lumacaftor improves the conformational stability of F508del-CFTR, resulting in increased processing and trafficking of mature protein to the cell surface.
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:
| 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 | cystic fibrosis transmembrane conductance regulator potentiators |
| CHEBI has role | CHEBI:71031 | orphan drugs |
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 | 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 |
| 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 | 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;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/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 |
| 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 |
| 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 |
| 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;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 |
| 125MG/PACKET;100MG/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, 2025 | NEW PATIENT POPULATION |
| 94MG/PACKET;75MG/PACKET | ORKAMBI | VERTEX PHARMS INC | N211358 | Sept. 2, 2022 | RX | GRANULE | ORAL | Sept. 2, 2025 | NEW STRENGTH |
| 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 |
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 | 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 | 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 |
| ORKAMBI | HUMAN PRESCRIPTION DRUG LABEL | 2 | 51167-809 | TABLET, FILM COATED | 200 mg | ORAL | NDA | 34 sections |
| ORKAMBI | HUMAN PRESCRIPTION DRUG LABEL | 2 | 51167-900 | GRANULE | 100 mg | ORAL | NDA | 34 sections |
| ORKAMBI | HUMAN PRESCRIPTION DRUG LABEL | 2 | 51167-900 | GRANULE | 100 mg | ORAL | NDA | 34 sections |
| ORKAMBI | HUMAN PRESCRIPTION DRUG LABEL | 2 | 51167-900 | GRANULE | 100 mg | ORAL | NDA | 34 sections |