Ivakaftor

Ivakaftor
Klinički podaci
Prodajno imeKalydeco
Podaci o licenci
Kategorija trudnoće
  • US: B (Bez rizika u ispitivanjima na životinjama)
Način primeneOralno
Pravni status
Pravni status
Identifikatori
CAS broj873054-44-5 Зелена квачицаДа
ATC kodNone
PubChemCID 16220172
ChemSpider17347474
UNII1Y740ILL1Z
SinonimiVX-770

Ivakaftor (Kalideko, VX-770) je lek za tretman cistične fibroze,[1] koji su razvili Verteks Farmaceutikals i Fondacija za cističnu fibrozu.[2] Ovo je privi lek prvi lek koji je odobren za lečenje uroka umesto simptoma bolesti.

Cistična fibroza je uzrokovana defektom proteina, cistično fibroznim regulatorom transmembranske provodnosti (CFTR), koji reguliše protok fluida unutar ćelija u utiče na komponente znoja, probavnih fluida, i sluzi. Defekt je uzrokovan DNK mutacijom koja se može javiti na nekoliko lokacija duž proteina, i svaka od njih utiče na nekoliko različitih funkcija proteina. Mutacija G551D, ne sprečava CFTR protein da dođe do površine epitelinih ćelija, ali mu onemogućava da transportuje hlor kroz jonski kanal. Ivakaftor poboljšava transport hlorida kroz jonski kanal. Ova vrsta leka je poznata kao CFTR potencijator.[3][4]

G551D je nađen kod oko 4-5% slučajeva cistične fibroze. Ivakaftor je efektivan (i odobren od strane FDA) samo za cističnu fibrozu sa tom mutacijom.[5] G551D je standardna aminokiselinska skraćenica za mutaciju kod koje je aminokiselina glicin (G) u poziciji 551 zamenjena sa aspartinskom kiselnom (D).

Kalideko će koštati $294,000 za godišnju opskrbu.[6]

Vidi još

Reference

  1. ^ Jones AM, Helm JM (2009). „Emerging treatments in cystic fibrosis”. Drugs. 69 (14): 1903—10. PMID 19747007. doi:10.2165/11318500-000000000-00000. 
  2. ^ Phase 3 Study of VX-770 Shows Marked Improvement in Lung Function Among People with Cystic Fibrosis with G551D Mutation, Feb. 23, 2011
  3. ^ Van Goor F, Hadida S, Grootenhuis PD, Burton B, Cao D, Neuberger T, Turnbull A, Singh A, Joubran J (2009). „Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770”. Proceedings of the National Academy of Sciences of the United States of America. 106 (44): 18825—30. PMC 2773991 Слободан приступ. PMID 19846789. doi:10.1073/pnas.0904709106. 
  4. ^ Sloane PA, Rowe SM (2010). „Cystic fibrosis transmembrane conductance regulator protein repair as a therapeutic strategy in cystic fibrosis”. Current opinion in pulmonary medicine. 16 (6): 591—7. PMID 20829696. doi:10.1097/MCP.0b013e32833f1d00. 
  5. ^ Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW (2010). „Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation”. The New England journal of medicine. 363 (21): 1991—2003. PMID 21083385. doi:10.1056/NEJMoa0909825. 
  6. ^ Drug Approved to Treat Cystic Fibrosis' Root Cause Архивирано на веб-сајту Wayback Machine (4. фебруар 2012) By MATTHEW PERRONE, AP Health Writer, ABC News, January 31, 2012

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