tezacaftor 🐶 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 | 5276 | 1152311-62-0 |
Description:
| Molecule | Description |
|---|---|
|
Synonyms:
|
Tezacaftor facilitates the cellular processing and trafficking of normal and select mutant forms of CFTR (including F508del-CFTR) to increase the amount of mature CFTR protein delivered to the cell surface.
Legend: OFP - off patent OFM - off market ONP - on patent |
Drug dosage:
None
ADMET properties:
| Property | Value | Reference |
|---|---|---|
| BDDCS (Biopharmaceutical Drug Disposition Classification System) | 2 | Bocci G, Oprea TI, Benet LZ |
Approvals:
| Date | Agency | Company | Orphan |
|---|---|---|---|
| Aug. 21, 2020 | EMA | Vertex Pharmaceuticals (Ireland) Limited | |
| Feb. 12, 2018 | FDA | VERTEX PHARMS |
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 | R07AX31 | RESPIRATORY SYSTEM OTHER RESPIRATORY SYSTEM PRODUCTS OTHER RESPIRATORY SYSTEM PRODUCTS Other respiratory system products |
| ATC | R07AX32 | RESPIRATORY SYSTEM OTHER RESPIRATORY SYSTEM PRODUCTS OTHER RESPIRATORY SYSTEM PRODUCTS Other respiratory system products |
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 | 12.64 | acidic |
| pKa2 | 13.25 | acidic |
| pKa3 | 13.99 | acidic |
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 |
|---|---|---|---|---|---|---|---|---|---|---|
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 8629162 | June 24, 2025 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF PATIENTS WITH A MILD TO MODERATE CLINICAL PHENOTYPE OF CYSTIC FIBROSIS HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 8629162 | June 24, 2025 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF PATIENTS WITH A MILD TO MODERATE CLINICAL PHENOTYPE OF CYSTIC FIBROSIS HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 8354427 | July 6, 2026 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE A R117H MUTATION IN THE CFTR GENE |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 8354427 | July 6, 2026 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE A R117H MUTATION IN THE CFTR GENE |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 9670163 | Dec. 28, 2026 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 9931334 | Dec. 28, 2026 | TREATING CYSTIC FIBROSIS PATIENTS AGES 12 AND OLDER, WHO ARE HOMOZYGOUS FOR F508DEL OR HAVE AT LEAST 1 CFTR GENE MUTATION RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (<30% CRYSTALLINE) IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 9931334 | Dec. 28, 2026 | TREATING CYSTIC FIBROSIS PATIENTS AGES 6 AND OLDER, WHO ARE HOMOZYGOUS FOR F508DEL OR HAVE AT LEAST 1 CFTR GENE MUTATION RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (<30% CRYSTALLINE) IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 9670163 | Dec. 28, 2026 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 9931334 | Dec. 28, 2026 | TREATING CYSTIC FIBROSIS PATIENTS AGES 12 AND OLDER, WHO ARE HOMOZYGOUS FOR F508DEL OR HAVE AT LEAST 1 CFTR GENE MUTATION RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (<30% CRYSTALLINE) IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 9931334 | Dec. 28, 2026 | TREATING CYSTIC FIBROSIS PATIENTS AGES 6 AND OLDER, WHO ARE HOMOZYGOUS FOR F508DEL OR HAVE AT LEAST 1 CFTR GENE MUTATION RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (<30% CRYSTALLINE) IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10022352 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR F508DEL AND A SECOND CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10022352 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR F508DEL AND A SECOND CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10239867 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10239867 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 11639347 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 9974781 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 9974781 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10022352 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR F508DEL AND A SECOND CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10022352 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR F508DEL AND A SECOND CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10239867 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10239867 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 11639347 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 9974781 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 9974781 | April 9, 2027 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 8598181 | May 1, 2027 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 8598181 | May 1, 2027 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 8324242 | Aug. 5, 2027 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 8324242 | Aug. 5, 2027 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 8415387 | Nov. 12, 2027 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 8415387 | Nov. 12, 2027 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 11564916 | Aug. 13, 2029 | TREATMENT OF CF IN A PATIENT AGE 6 YEARS AND OLDER WHO IS HOMOZYGOUS FOR F508DEL OR HAS AT LEAST ONE CFTR GENE MUTATION RESPONSIVE TO TEZ/IVA BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE USING THE COMPOSITION RECITED IN US 11564916 CLAIM 1 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 11564916 | Aug. 13, 2029 | TREATMENT OF CF IN A PATIENT AGE 6 YEARS AND OLDER WHO IS HOMOZYGOUS FOR F508DEL OR HAS AT LEAST ONE CFTR GENE MUTATION RESPONSIVE TO TEZ/IVA BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE USING THE COMPOSITION RECITED IN US 11564916 CLAIM 1 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10081621 | March 25, 2031 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10081621 | March 25, 2031 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10081621 | March 25, 2031 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10081621 | March 25, 2031 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 11578062 | March 25, 2031 | TREATMENT OF CF IN A PATIENT AGE 6 YEARS AND OLDER WHO IS HOMOZYGOUS FOR F508DEL OR HAS AT LEAST ONE CFTR GENE MUTATION RESPONSIVE TO TEZ/IVA BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE USING THE COMPOSITION RECITED IN US 11578062 CLAIM 6 OR 13 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10081621 | March 25, 2031 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10081621 | March 25, 2031 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10081621 | March 25, 2031 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10081621 | March 25, 2031 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 11578062 | March 25, 2031 | TREATMENT OF CF IN A PATIENT AGE 6 YEARS AND OLDER WHO IS HOMOZYGOUS FOR F508DEL OR HAS AT LEAST ONE CFTR GENE MUTATION RESPONSIVE TO TEZ/IVA BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE USING THE COMPOSITION RECITED IN US 11578062 CLAIM 6 OR 13 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10058546 | July 15, 2033 | TREATMENT OF CF IN PATIENTS 12 YEARS AND OLDER WHO HAVE A F508DEL OR G551D CFTR MUTATION AND A 2ND MUTATION SELECTED FROM R117H, A455E, 2789+5G->A, & 3849+10KBC->T, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF US 10058546 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10058546 | July 15, 2033 | TREATMENT OF CF IN PATIENTS 6 YEARS AND OLDER WHO HAVE A F508DEL OR G551D CFTR MUTATION AND A 2ND MUTATION SELECTED FROM R117H, A455E, 2789+5G->A, AND 3849+10KBC->T, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF US 10058546 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10058546 | July 15, 2033 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 12 YEARS AND OLDER, WITH A F508DEL OR G551D CFTR GENE MUTATION AND A A455E, 2789+5G->A, OR 3849+10KBC->T MUTATION, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF U.S PATENT 10058546 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10058546 | July 15, 2033 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 6 YEARS AND OLDER, WITH A F508DEL OR G551D CFTR GENE MUTATION AND A A455E, 2789+5G->, OR 3849+10KBC->T MUTATION, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF U.S. PATENT 10058546 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 9012496 | July 15, 2033 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR THE F508DEL MUTATION AND A SECOND MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10058546 | July 15, 2033 | TREATMENT OF CF IN PATIENTS 12 YEARS AND OLDER WHO HAVE A F508DEL OR G551D CFTR MUTATION AND A 2ND MUTATION SELECTED FROM R117H, A455E, 2789+5G->A, & 3849+10KBC->T, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF US 10058546 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10058546 | July 15, 2033 | TREATMENT OF CF IN PATIENTS 6 YEARS AND OLDER WHO HAVE A F508DEL OR G551D CFTR MUTATION AND A 2ND MUTATION SELECTED FROM R117H, A455E, 2789+5G->A, AND 3849+10KBC->T, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF US 10058546 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10058546 | July 15, 2033 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 12 YEARS AND OLDER, WITH A F508DEL OR G551D CFTR GENE MUTATION AND A A455E, 2789+5G->A, OR 3849+10KBC->T MUTATION, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF U.S PATENT 10058546 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10058546 | July 15, 2033 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 6 YEARS AND OLDER, WITH A F508DEL OR G551D CFTR GENE MUTATION AND A A455E, 2789+5G->, OR 3849+10KBC->T MUTATION, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF U.S. PATENT 10058546 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 9012496 | July 15, 2033 | TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR THE F508DEL MUTATION AND A SECOND MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10206877 | April 14, 2035 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10206877 | April 14, 2035 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10206877 | April 14, 2035 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877 |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | 10206877 | April 14, 2035 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10206877 | April 14, 2035 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10206877 | April 14, 2035 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10206877 | April 14, 2035 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | 10206877 | April 14, 2035 | TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877 |
Orange Book exclusivity data (new drug applications)
| Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Exclusivity date | Description |
|---|---|---|---|---|---|---|---|---|---|
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | Feb. 12, 2023 | NEW CHEMICAL ENTITY |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | Feb. 12, 2023 | NEW CHEMICAL ENTITY |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | Feb. 12, 2025 | TREATMENT OF PATIENTS WITH CYSTIC FIBROSIS AGED 12 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR GENE RESPONSIVE TO TEZACAFTOR/IVACAFTOR |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | Feb. 12, 2025 | TREATMENT OF PATIENTS WITH CYSTIC FIBROSIS AGED 12 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR GENE RESPONSIVE TO TEZACAFTOR/IVACAFTOR |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | June 21, 2026 | TX OF PTS W/ CYSTIC FIBROSIS (CF) AGE 6 TO <12 YRS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR W/ AT LEAST 1 MUTATION IN CF TRANSMEMBRANE CONDUCTANCE REGULATORY GENE RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | June 21, 2026 | TX OF PTS W/ CYSTIC FIBROSIS (CF) AGE 6 TO <12 YRS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR W/ AT LEAST 1 MUTATION IN CF TRANSMEMBRANE CONDUCTANCE REGULATORY GENE RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE |
| 150MG;150MG, 100MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | Feb. 12, 2018 | RX | TABLET | ORAL | Dec. 21, 2027 | FOR TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE AT LEAST ONE OF THE ADDITIONAL MUTATIONS IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT HAVE BEEN IDENTIFIED AS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND IDENTIFIED IN THE APPROVAL ON DECEMBER 21, 2020 |
| 75MG;75MG, 50MG | SYMDEKO (COPACKAGED) | VERTEX PHARMS INC | N210491 | June 21, 2019 | RX | TABLET | ORAL | Dec. 21, 2027 | FOR TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE AT LEAST ONE OF THE ADDITIONAL MUTATIONS IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT HAVE BEEN IDENTIFIED AS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND IDENTIFIED IN THE APPROVAL ON DECEMBER 21, 2020 |
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 | 6.57 | CHEMBL | DRUG LABEL |
External reference:
| ID | Source |
|---|---|
| 8RW88Y506K | UNII |
| C4519194 | UMLSCUI |
| CV6 | PDB_CHEM_ID |
| CHEMBL3544914 | ChEMBL_ID |
| 46199646 | PUBCHEM_CID |
| DB11712 | DRUGBANK_ID |
| D11041 | KEGG_DRUG |
| 10104 | INN_ID |
| 10199 | IUPHAR_LIGAND_ID |
| 33763 | MMSL |
| d08742 | MMSL |
| 017465 | NDDF |
| 771596001 | SNOMEDCT_US |
| 4037327 | VANDF |
| 1999382 | RXNORM |
| C000625213 | MESH_SUPPLEMENTAL_RECORD_UI |
Pharmaceutical products:
None