> Trialight public data. Index of pages: https://trialight.com/llms.txt
> Web page: https://trialight.com/data/asset/tezacaftor-361d103a-bfda-4757-93c8-8092efda7716
> Record changed: 2026-10-13

# Tezacaftor

Highest phase reached

Approved

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

Drug type

Small molecule

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

First approval year

2018

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

## Development status

**First approval year**

2018

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

**Listing at source**

Listed

**Highest phase reached**

Approved

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

## Names and chemistry

**Drug type**

Small molecule

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

**Drug name**

Tezacaftor

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

**Molecular weight**

520.5

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

**Structure (SMILES)**

CC(C)(CO)c1cc2cc(NC(=O)C3(c4ccc5c(c4)OC(F)(F)O5)CC3)c(F)cc2n1C\[C@@H\](O)CO

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

### Brand names (4)

- tezacaftor component of symkevi
- Tezacaftor component of symkevi
- tezacaftor component of trikafta
- Tezacaftor component of trikafta

### Other names (19)

- 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

## Identifiers

**CAS registry numbers**

- 1152311-62-0

**ChEMBL ID**

CHEMBL3544914

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

**InChIKey**

MJUVRTYWUMPBTR-MRXNPFEDSA-N

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3544914)

**PubChem compound IDs**

- 46199646

**RxNorm IDs (RxCUI)**

- 1999382

**FDA ingredient codes (UNII)**

- 8RW88Y506K

## Regulatory information

### United States

**Applicant**

VERTEX PHARMS INC

**Approval date**

20 Dec 2024

**Application number**

NDA218730

### Other regulatory facts

**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

### Drug classification

**Pharmacology name**

tezacaftor

**Type**

Synthetic organic

**Drug name stem**

\-caftor

**Approval recorded by Guide to Pharmacology**

Yes

#### Administration routes

- Oral

### Mechanisms of action (2)

- **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

### Open Targets indications (9)

- **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

### Reported adverse events (39)

- **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

And 14 more, listed on the interactive page.

### Trial references (55)

- NCT01531673  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT01531673)
- NCT02015507  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02015507)
- NCT02070744  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02070744)
- NCT02347657  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02347657)
- NCT02392234  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02392234)
- NCT02412111  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02412111)
- NCT02508207  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02508207)
- NCT02516410  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02516410)
- NCT02565914  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02565914)
- NCT02730208  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02730208)
- NCT02951195  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02951195)
- NCT02953314  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02953314)
- NCT03029455  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03029455)
- NCT03150719  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03150719)
- NCT03224351  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03224351)
- NCT03227471  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03227471)
- NCT03447249  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03447249)
- NCT03447262  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03447262)
- NCT03460990  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03460990)
- NCT03525444  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03525444)
- NCT03525548  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03525548)
- NCT03525574  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03525574)
- NCT03537651  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03537651)
- NCT03559062  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03559062)
- NCT03624101  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03624101)

And 30 more, listed on the interactive page.

## Dosing

**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

## Chemical properties

**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

## National Drug Codes

- 51167-121
- 51167-135

## Evidence counts

**Recorded assays**

97

**Active assays**

5

### Active assay types

- Confirmatory

## Biological measurements

### Observed measurements

#### 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](https://trialight.com/data/trial/evaluation-of-vx-828-in-healthy-participants-and-in-7648273e-ae83-4332-a1e0-005b2100ed65.md)
- 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)

- [Cystic fibrosis](https://trialight.com/data/condition/cystic-fibrosis-6f9870eb-6ebc-5bec-a50b-34b0b323a7c3.md)

### Other linked conditions (1)

- [Acute lung injury](https://trialight.com/data/condition/acute-lung-injury-fb11c447-d7f6-5c24-8fb9-764bbaaabced.md)

### Targets (1)

- [CFTR](https://trialight.com/data/target/cftr-1582a227-49f3-5e47-a81a-1acc4b67f420.md) Cystic fibrosis transmembrane conductance regulator positive modulator

---

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