Tezacaftor
Highest phase reached
Approved
Drug type
Small molecule
First approval year
2018
- First approval year
- 2018
- Listing at source
- Listed
- Highest phase reached
- Approved
- Drug type
- Small molecule
- Drug name
- Tezacaftor
- Molecular weight
- 520.5
- Structure (SMILES)
- CC(C)(CO)c1cc2cc(NC(=O)C3(c4ccc5c(c4)OC(F)(F)O5)CC3)c(F)cc2n1C[C@@H](O)CO
- tezacaftor component of symkevi
- Tezacaftor component of symkevi
- tezacaftor component of trikafta
- Tezacaftor component of trikafta
- elexacaftor ivacaftor tezacaftor
- symkevi component tezacaftor
- SYMKEVI COMPONENT TEZACAFTOR
- tezacaftor
- Tezacaftor
- tezacaftor [INN]
- TEZACAFTOR [MI]
- TEZACAFTOR [ORANGE BOOK]
- TEZACAFTOR [USAN]
- Tezacaftor [WHO-DD]
- tezacaftor inn
- tezacaftor mi
- tezacaftor orange book
- tezacaftor usan
- tezacaftor who dd
- trikafta component tezacaftor
- TRIKAFTA COMPONENT TEZACAFTOR
- vx 661
- VX-661
- CAS registry numbers
- 1152311-62-0
- ChEMBL ID
- CHEMBL3544914
- InChIKey
- MJUVRTYWUMPBTR-MRXNPFEDSA-N
- PubChem compound IDs
- 46199646
- RxNorm IDs (RxCUI)
- 1999382
- FDA ingredient codes (UNII)
- 8RW88Y506K
- Applicant
- VERTEX PHARMS INC
- Approval date
- 20 Dec 2024
- Application number
- NDA218730
- Patents
- 0
- Designations
- 1
- First in class
- No
- Fast track
- No
- Breakthrough designation
- No
- Orphan designation
- No
- Priority review
- No
- Orphan exclusivity
- No
- Accelerated approval
- No
- Availability
- Prescription only
- Pharmacology name
- tezacaftor
- Type
- Synthetic organic
- Drug name stem
- -caftor
- Approval recorded by Guide to Pharmacology
- Yes
Administration routes
- Oral
- Action
- Activator
- Action type
- ACTIVATOR
- Target
- CFTR
- Gene symbol
- CFTR
- Protein identifier
- P13569
- Median affinity
- 6.57
- Affinity units
- pEC50
- Mechanism
- Cystic fibrosis transmembrane conductance regulator positive modulator
- Action type
- POSITIVE_MODULATOR
- Target
- Cystic fibrosis transmembrane conductance regulator
- Protein identifier
- P13569
Open Targets mechanisms
- Mechanism
- Cystic fibrosis transmembrane conductance regulator positive modulator
- Action type
- POSITIVE MODULATOR
- Target
- Cystic fibrosis transmembrane conductance regulator
DrugCentral mechanisms
- Action type
- PHARMACOLOGICAL CHAPERONE
- Gene symbol
- CFTR
- Protein identifier
- P13569
- Organism
- Homo sapiens
- Target development level
- Clinical
Investigated indications
- Condition
- Cystic Fibrosis
- Highest recorded phase
- 4
- Condition identifier
- MONDO:0009061
- Subject identifier
- D003550
- Condition identifier
- EFO_0002546
- Condition identifier
- EFO_0004610
- Condition identifier
- EFO_0801077
- Highest recorded phase
- 3
- Condition identifier
- HP_0002094
- Condition identifier
- HP_0040270
- Highest recorded phase
- 3
- Condition identifier
- MONDO_0006031
- Condition identifier
- MONDO_0009061
- Condition identifier
- MONDO_0009061
- Highest recorded phase
- 4
- Condition identifier
- MONDO_0015796
- Highest recorded phase
- 4
- Event
- anaemia
- Reports
- 2
- Reporting signal score
- 35.4087
- Event
- arthralgia
- Reports
- 12
- Reporting signal score
- 27.5797
- Event
- asthenia
- Reports
- 8
- Reporting signal score
- 33.3429
- Event
- back pain
- Reports
- 4
- Reporting signal score
- 19.9342
- Event
- cerebrovascular accident
- Reports
- 1
- Reporting signal score
- 21.1634
- Event
- cough
- Reports
- 91
- Reporting signal score
- 23.7268
- Event
- cystic fibrosis
- Reports
- 109
- Reporting signal score
- 462.588
- Event
- cystic fibrosis lung
- Reports
- 5
- Reporting signal score
- 21.546
- Event
- cystic fibrosis respiratory infection suppression
- Reports
- 73
- Reporting signal score
- 412.263
- Event
- diarrhoea
- Reports
- 24
- Reporting signal score
- 44.4097
- Event
- distal intestinal obstruction syndrome
- Reports
- 8
- Reporting signal score
- 30.1232
- Event
- drug hypersensitivity
- Reports
- 10
- Reporting signal score
- 23.3635
- Event
- fall
- Reports
- 5
- Reporting signal score
- 38.0666
- Event
- fatigue
- Reports
- 48
- Reporting signal score
- 22.3462
- Event
- forced expiratory volume decreased
- Reports
- 30
- Reporting signal score
- 85.9841
- Event
- gait disturbance
- Reports
- 1
- Reporting signal score
- 19.7449
- Event
- haemoptysis
- Reports
- 79
- Reporting signal score
- 145.083
- Event
- hospitalisation
- Reports
- 260
- Reporting signal score
- 405.358
- Event
- hypertension
- Reports
- 1
- Reporting signal score
- 32.3301
- Event
- hypotension
- Reports
- 1
- Reporting signal score
- 38.5547
- Event
- infection
- Reports
- 199
- Reporting signal score
- 264.628
- Event
- infective pulmonary exacerbation of cystic fibrosis
- Reports
- 765
- Reporting signal score
- 4,195.96
- Event
- insurance issue
- Reports
- 12
- Reporting signal score
- 24.3558
- Event
- liver function test increased
- Reports
- 35
- Reporting signal score
- 40.0253
- Event
- lung infection
- Reports
- 34
- Reporting signal score
- 49.0392
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And 30 more, listed on the interactive page.
- Dose
- Two tablets of vanzacaftor 10 mg/tezacaftor 50 mg/deutivacaftor 125 mg in patients 12 years and older or two tablets of vanzacaftor 4 mg/tezacaftor 20 mg/deutivacaftor 50 mg in patients 6 to less than 12 years old
- Route
- Oral
- How often
- Once daily
- Hydrogen bond acceptors
- 7
- Hydrogen bond donors
- 4
- Polar surface area
- 113.18
- Calculated partition coefficient (ALogP)
- 3.4
- Calculated partition coefficient (XLogP)
- 2.9
- Prodrug
- No
- Chirality
- Single enantiomer
- Inorganic
- No
- Rule-of-five violations
- 1
- Rotatable bonds
- 8
- Heavy atoms
- 37
- Molecular formula
- C26H27F3N2O6
- Monoisotopic mass
- 520.18212107
- Natural product
- No
- 51167-121
- 51167-135
- Recorded assays
- 97
- Active assays
- 5
Active assay types
- Confirmatory
Oral bioavailability
- Median
- 50
- Mean
- 50
- Minimum
- 50
- Maximum
- 50
- Unit
- %
- Assays
- 1
- Standard deviation
- 0
- Summary method
- median
- Censored results excluded
- 0
- Other units excluded
- 0
- Unknown units excluded
- 0
Measurement types
- F
Related records
Trials (22)
- A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
- A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor
- A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation
- A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
- A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis
- A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)
- A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
- A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
- An Open-Label, Phase 1 Study in Healthy Adult Subjects to Examine the Effects of Multiple-Dose Ciprofloxacin on Ivacaftor and VX-661 in Combination With Ivacaftor
- Daily Monitoring of Respiratory Symptoms and Spirometry During ETI Treatment in Persons With Cystic Fibrosis.
- Effect of the CFTR-modulating Triple Therapy Elexacaftor - Tezacaftor - Ivacaftor
- Ensuring Access to Optimal Therapy in CF: The ENACT Study
- Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis
- Gut Imaging for Function & Transit in Cystic Fibrosis Study 2
- Gut Imaging for Function and Transit in Cystic Fibrosis 3 Junior
- Impact of Elexacaftor-Tezacaftor-Ivacaftor Treatment on Metabolic, Epigenetic and Fecal Microbiota Profiles in People With Cystic Fibrosis.
- Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators
- Sinus Disease in Young Children With Cystic Fibrosis
- Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
- Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Approved for (1)
Other linked conditions (1)
Targets (1)
- CFTR Cystic fibrosis transmembrane conductance regulator positive modulator