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1. 3-pyridinecarboxamide, N-((1,3-dimethyl-1h-pyrazol-4-yl)sulfonyl)-6-(3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1h-pyrazol-1-yl)-2-((4s)-2,2,4-trimethyl-1-pyrrolidinyl)-
2. Vx-445
3. Vx445
1. 2216712-66-0
2. Vx-445
3. Elexacaftor [usan]
4. Rrn67gmb0v
5. Elexacaftor (usan)
6. (s)-n-((1,3-dimethyl-1h-pyrazol-4-yl)sulfonyl)-6-(3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1h-pyrazol-1-yl)-2-(2,2,4-trimethylpyrrolidin-1-yl)nicotinamide
7. 3-pyridinecarboxamide, N-((1,3-dimethyl-1h-pyrazol-4-yl)sulfonyl)-6-(3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1h-pyrazol-1-yl)-2-((4s)-2,2,4-trimethyl-1-pyrrolidinyl)-
8. N-(1,3-dimethylpyrazol-4-yl)sulfonyl-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)pyrazol-1-yl]-2-[(4s)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide
9. (6p)-n-(1,3-dimethyl-1h-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1h-pyrazol-1-yl]-2-[(4s)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide
10. Wjx
11. Elexacaftor [mi]
12. Unii-rrn67gmb0v
13. Elexacaftor [inn]
14. Elexacaftor (vx-445)
15. Elexacaftor [who-dd]
16. Elexacaftor/ivacaftor/tezacaftor
17. Chembl4298128
18. Schembl20239811
19. Gtpl10552
20. Elexacaftor [orange Book]
21. Dtxsid901027907
22. Ex-a3637
23. S8851
24. Trikafta Component Elexacaftor
25. Vx-445vx-445
26. Who 11180
27. At16051
28. Db15444
29. Elexacaftor Component Of Trikafta
30. Compound 1 [wo2018107100a1]
31. Ac-36746
32. Hy-111772
33. Cs-0090942
34. D11507
35. A930250
Molecular Weight | 597.7 g/mol |
---|---|
Molecular Formula | C26H34F3N7O4S |
XLogP3 | 4.9 |
Hydrogen Bond Donor Count | 1 |
Hydrogen Bond Acceptor Count | 11 |
Rotatable Bond Count | 8 |
Exact Mass | 597.23450825 g/mol |
Monoisotopic Mass | 597.23450825 g/mol |
Topological Polar Surface Area | 133 Ų |
Heavy Atom Count | 41 |
Formal Charge | 0 |
Complexity | 1050 |
Isotope Atom Count | 0 |
Defined Atom Stereocenter Count | 1 |
Undefined Atom Stereocenter Count | 0 |
Defined Bond Stereocenter Count | 0 |
Undefined Bond Stereocenter Count | 0 |
Covalently Bonded Unit Count | 1 |
Elexacaftor, in combination with [ivacaftor] and [tezacaftor] as the combination product TrikaftaTM, is indicated for the treatment of cystic fibrosis (CF) in patients 12 years of age and older who have at least one _F508del_ mutation in the CTFR gene.
As a CFTR corrector, elexacaftor works to increase the amount of mature CFTR proteins present on the surface of cells. When used in combination with CFTR potentiators, which enhance the function of cell-surface CFTR proteins, drugs like elexacaftor help to improve a variety of multi-organ cystic fibrosis symptoms, including lung function, nutritional status, and overall quality of life. TrikaftaTM, the triple combination product containing elexacaftor, may cause elevations in liver transaminases. Liver function testing should be conducted prior to beginning Trikafta, every 3 months for the first year of treatment, and annually thereafter.
Chloride Channel Agonists
A class of drugs that stimulate chloride ion influx through cell membrane channels. (See all compounds classified as Chloride Channel Agonists.)
Absorption
The absolute oral bioavailability of elexacaftor is approximately 80%. The steady-state AUC0-24h and Cmax following once daily dosing with elexacaftor 200mg are 162 mcgh/mL and 8.7 mcg/mL, respectively, and the median Tmax is 6 hours. The AUC of elexacaftor is increased 1.9-2.5-fold following a moderate-fat meal - for this reason, it is recommended to give TrikaftaTM with fat-containing food.
Route of Elimination
Approximately 87.3% of an administered radio-labeled dose of elexacaftor was found in the feces, mostly as metabolites, while only 0.23% of that same dose was found excreted in the urine.
Volume of Distribution
The apparent volume of distribution of elexacaftor is 53.7 L.
Clearance
The mean apparent clearance of elexacaftor is 1.18 L/h.
The metabolism of elexacaftor is extensive and primarily catalyzed via CYP3A4/5. Its main active metabolite, M23-ELX, carries a similar potency as the parent drug. The precise metabolic pathway of elexacaftor has not yet been elucidated in published research.
The mean terminal half-life of elexacaftor is approximately 24.7 hours.
Cystic fibrosis (CF) is the result of a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR proteins produced by this gene are transmembrane ion channels that move sodium and chloride across cell membranes - water follows the flow of chloride ions to the cell surface, which consequently helps to hydrate the surface of the cell and thin the secretions (i.e. mucous) around the cell. Mutations in the CFTR gene produce CFTR proteins of insufficient quantity and/or function, leading to defective ion transport and a build-up of thick mucous throughout the body that causes multi-organ disease involving the pulmonary, gastrointestinal, and pancreatic systems (amongst others). The most common CFTR mutation, the _F508del_ mutation, is estimated to account for 70 to 90% of all CFTR mutations and results in severe processing and trafficking defects of the CFTR protein. Elexacaftor is a CFTR corrector that modulates CFTR proteins to facilitate trafficking to the cell surface for incorporation into the cell membrane. The end result is an increase in the number of mature CFTR proteins present at the cell surface and, therefore, improved ion transport and CF symptomatology. Elexacaftor is used in combination with tezacaftor, another CFTR corrector with a different mechanism of action, and ivacaftor, a CFTR potentiator that improves the function of CFTR proteins on the cell surface - this multi-faceted, triple-drug approach confers a synergistic effect beyond that seen in typical corrector/potentiator dual therapy regimens.
https://www.pharmacompass.com/radio-compass-blog/new-drugs-approvals-by-fda-and-ema-2020-recap
Global Sales Information
Main Therapeutic Indication : Genetic Disorders
Currency : USD
2019 Revenue in Millions : 420
2018 Revenue in Millions : 0
Growth (%) : New launch
Main Therapeutic Indication : Genetic Disorders
Currency : USD
2020 Revenue in Millions : 3,864
2019 Revenue in Millions : 420
Growth (%) : 820
Main Therapeutic Indication : Genetic Disorders
Currency : USD
2021 Revenue in Millions : 5,697
2020 Revenue in Millions : 3,864
Growth (%) : 32
Main Therapeutic Indication : Genetic Diseases
Currency : USD
2022 Revenue in Millions : 7,687
2021 Revenue in Millions : 5,697
Growth (%) : 35
Main Therapeutic Indication : Genetic Diseases
Currency : USD
2023 Revenue in Millions : 8,945
2022 Revenue in Millions : 7,687
Growth (%) : 14
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ABOUT THIS PAGE
A Elexacaftor manufacturer is defined as any person or entity involved in the manufacture, preparation, processing, compounding or propagation of Elexacaftor, including repackagers and relabelers. The FDA regulates Elexacaftor manufacturers to ensure that their products comply with relevant laws and regulations and are safe and effective to use. Elexacaftor API Manufacturers are required to adhere to Good Manufacturing Practices (GMP) to ensure that their products are consistently manufactured to meet established quality criteria.
click here to find a list of Elexacaftor manufacturers with USDMF, JDMF, KDMF, CEP, GMP, COA and API Price related information on PhamaCompass.
A Elexacaftor supplier is an individual or a company that provides Elexacaftor active pharmaceutical ingredient (API) or Elexacaftor finished formulations upon request. The Elexacaftor suppliers may include Elexacaftor API manufacturers, exporters, distributors and traders.
click here to find a list of Elexacaftor suppliers with USDMF, JDMF, KDMF, CEP, GMP, COA and API Price related information on PharmaCompass.
National Drug Code is a comprehensive database maintained by the FDA that contains information on all drugs marketed in the US. This directory includes information about finished drug products, unfinished drug products, and compounded drug products, including those containing Elexacaftor as an active pharmaceutical ingredient (API).
The FDA updates the NDC directory daily. The NDC numbers for Elexacaftor API and other APIs are published in this directory by the FDA.
The NDC unfinished drugs database includes product listing information submitted for all unfinished drugs, such as active pharmaceutical ingredients (APIs), drugs intended for further processing and bulk drug substances for compounding.
Pharmaceutical companies that manufacture Elexacaftor as an active pharmaceutical ingredient (API) must furnish the FDA with an updated record of all drugs that they produce, prepare, propagate, compound, or process for commercial distribution in the US at their facilities.
The NDC directory also contains data on finished compounded human drug products that contain Elexacaftor and are produced by outsourcing facilities. While these outsourcing facilities are not mandated to assign a Elexacaftor NDC to their finished compounded human drug products, they may choose to do so.
click here to find a list of Elexacaftor suppliers with NDC on PharmaCompass.
Elexacaftor Active pharmaceutical ingredient (API) is produced in GMP-certified manufacturing facility.
GMP stands for Good Manufacturing Practices, which is a system used in the pharmaceutical industry to make sure that goods are regularly produced and monitored in accordance with quality standards. The FDA’s current Good Manufacturing Practices requirements are referred to as cGMP or current GMP which indicates that the company follows the most recent GMP specifications. The World Health Organization (WHO) has its own set of GMP guidelines, called the WHO GMP. Different countries can also set their own guidelines for GMP like China (Chinese GMP) or the EU (EU GMP).
PharmaCompass offers a list of Elexacaftor GMP manufacturers, exporters & distributors, which can be sorted by USDMF, JDMF, KDMF, CEP (COS), WC, API price, and more, enabling you to easily find the right Elexacaftor GMP manufacturer or Elexacaftor GMP API supplier for your needs.
A Elexacaftor CoA (Certificate of Analysis) is a formal document that attests to Elexacaftor's compliance with Elexacaftor specifications and serves as a tool for batch-level quality control.
Elexacaftor CoA mostly includes findings from lab analyses of a specific batch. For each Elexacaftor CoA document that a company creates, the USFDA specifies specific requirements, such as supplier information, material identification, transportation data, evidence of conformity and signature data.
Elexacaftor may be tested according to a variety of international standards, such as European Pharmacopoeia (Elexacaftor EP), Elexacaftor JP (Japanese Pharmacopeia) and the US Pharmacopoeia (Elexacaftor USP).
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