Stem definition | Drug id | CAS RN |
---|---|---|
cystic fibrosis transmembrane regulator (CFTR) protein modulators | 5372 | 2216712-66-0 |
None
None
Date | Agency | Company | Orphan |
---|---|---|---|
Aug. 21, 2020 | EMA | Vertex Pharmaceuticals (Ireland) Limited | |
Oct. 10, 2019 | FDA | VERTEX PHARMS INC |
MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
---|---|---|---|---|---|---|
Infective pulmonary exacerbation of cystic fibrosis | 464.51 | 40.72 | 114 | 3042 | 10693 | 46672213 |
Productive cough | 218.66 | 40.72 | 93 | 3063 | 50622 | 46632284 |
Headache | 139.10 | 40.72 | 165 | 2991 | 478187 | 46204719 |
Cough | 116.19 | 40.72 | 107 | 3049 | 230142 | 46452764 |
Abdominal pain upper | 111.34 | 40.72 | 86 | 3070 | 145219 | 46537687 |
Hospitalisation | 84.97 | 40.72 | 53 | 3103 | 63332 | 46619574 |
Influenza | 84.31 | 40.72 | 60 | 3096 | 89210 | 46593696 |
Rash | 71.71 | 40.72 | 103 | 3053 | 356409 | 46326497 |
Haemoptysis | 71.39 | 40.72 | 35 | 3121 | 26154 | 46656752 |
Cystic fibrosis | 59.33 | 40.72 | 18 | 3138 | 3670 | 46679236 |
Sputum increased | 57.74 | 40.72 | 15 | 3141 | 1741 | 46681165 |
Weight increased | 54.35 | 40.72 | 61 | 3095 | 164412 | 46518494 |
Paranasal sinus hypersecretion | 53.00 | 40.72 | 16 | 3140 | 3208 | 46679698 |
Exposure during pregnancy | 46.13 | 40.72 | 46 | 3110 | 108166 | 46574740 |
Distal intestinal obstruction syndrome | 43.38 | 40.72 | 9 | 3147 | 388 | 46682518 |
MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
---|---|---|---|---|---|---|
Infective pulmonary exacerbation of cystic fibrosis | 332.51 | 51.27 | 84 | 2034 | 8488 | 29941872 |
Productive cough | 259.01 | 51.27 | 92 | 2026 | 29861 | 29920499 |
Headache | 143.20 | 51.27 | 111 | 2007 | 182195 | 29768165 |
Abdominal pain upper | 131.57 | 51.27 | 71 | 2047 | 62480 | 29887880 |
Cough | 103.69 | 51.27 | 79 | 2039 | 125563 | 29824797 |
Influenza | 73.97 | 51.27 | 43 | 2075 | 43355 | 29907005 |
Sputum increased | 70.78 | 51.27 | 18 | 2100 | 1839 | 29948521 |
Testicular pain | 68.19 | 51.27 | 19 | 2099 | 2753 | 29947607 |
Weight increased | 54.80 | 51.27 | 44 | 2074 | 74869 | 29875491 |
Paranasal sinus hypersecretion | 52.50 | 51.27 | 12 | 2106 | 780 | 29949580 |
Source | Code | Description |
---|---|---|
ATC | R07AX32 | RESPIRATORY SYSTEM OTHER RESPIRATORY SYSTEM PRODUCTS OTHER RESPIRATORY SYSTEM PRODUCTS Other respiratory system products |
MeSH PA | D065101 | Chloride Channel Agonists |
MeSH PA | D049990 | Membrane Transport Modulators |
Disease | Relation | SNOMED_ID | DOID |
---|---|---|---|
Cystic fibrosis | indication | 190905008 | DOID:1485 |
Dissociation level | Dissociation constant | Type (acidic/basic) |
---|---|---|
pKa1 | 4.64 | acidic |
pKa2 | 1.88 | Basic |
None
Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Exclusivity date | Description |
---|---|---|---|---|---|---|---|---|---|
100MG,75MG,50MG;N/A,150MG,N/A | TRIKAFTA (COPACKAGED) | VERTEX PHARMS INC | N212273 | Oct. 21, 2019 | RX | TABLET, TABLET | ORAL | Feb. 12, 2023 | NEW CHEMICAL ENTITY |
100MG,75MG,50MG;N/A,150MG,N/A | TRIKAFTA (COPACKAGED) | VERTEX PHARMS INC | N212273 | Oct. 21, 2019 | RX | TABLET, TABLET | ORAL | Oct. 21, 2024 | NEW CHEMICAL ENTITY |
100MG,75MG,50MG;N/A,150MG,N/A | TRIKAFTA (COPACKAGED) | VERTEX PHARMS INC | N212273 | Oct. 21, 2019 | RX | TABLET, TABLET | ORAL | Oct. 21, 2026 | INDICATED FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 12 YEARS AND OLDER WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE |
100MG,75MG,50MG;N/A,150MG,N/A | TRIKAFTA (COPACKAGED) | VERTEX PHARMS INC | N212273 | Oct. 21, 2019 | RX | TABLET, TABLET | ORAL | Dec. 21, 2027 | TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 12 YEARS AND OLDER WHO HAVE A MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA |
Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
---|---|---|---|---|---|---|---|---|---|
Cystic fibrosis transmembrane conductance regulator | Ion channel | UNKNOWN | DRUG LABEL |
ID | Source |
---|---|
4038884 | VANDF |
C5139717 | UMLSCUI |
CHEMBL4298128 | ChEMBL_ID |
DB15444 | DRUGBANK_ID |
D11700 | KEGG_DRUG |
C000629074 | MESH_SUPPLEMENTAL_RECORD_UI |
134587348 | PUBCHEM_CID |
10552 | IUPHAR_LIGAND_ID |
11180 | INN_ID |
RRN67GMB0V | UNII |
2256951 | RXNORM |
37631 | MMSL |
d09420 | MMSL |
018170 | NDDF |
827070004 | SNOMEDCT_US |
None