Ivacaftor: First Novel CFTR Potentiator Drug for Rare Cystic Fibrosis Imran Mohammed*,**,1, Ahmed Tanveer***, Tabrez Shams****,** *Assistant Professor, Department of Pharmacology, HIMSR **Associated, Hakeem Abdul Hameed Centenary Hospital, Jamia Hamdard, New Delhi-110062 ***Assistant Professor, Department of Anatomy, Faculty of Dentistry, Jamia Millia Islamia, New Delhi-110025 ****Assistant Professor, Department of Physiology, HIMSR 1Corresponding Author Email: drimran@aol.in
Online published on 27 March, 2014. Abstract Cystic Fibrosis (CF) is an autosomal recessive genetic disorder. It is caused by a mutation in Cystic Fibrosis Transmembrane Regulator (CFTR). This mutation results in chloride transporter defect thereby leading to thick mucus secretion in the lungs associated with breathing difficulties and secondary infections. Earlier managed by symptomatic treatment but now FDA has approved a new first-in-class novel CFTR potentiator drug called Ivacaftor. The related search was made on the PUBMED/MEDLINE with the word Cystic Fibrosis and Ivacaftor. Ivacaftor has added a new horizon to the CF treatment. It facilitates the chloride transport by potentiating the open channel probability of CFTR in G551D mutation only. It has been approved as 150 mg twice daily dose taken with the fatty meals in patients aged 6 years or older. It is metabolised through CYP3A4 generating two active metabolites. It is primarily excreted in the faeces with the terminal half-life of 12 hours. Although associated with the limitations but it will prove a better armament in the management of Cystic Fibrosis patients having G551D mutation as a personalised medicine approach. Top Keywords Ivacaftor, CFTR potentiator, Cystic Fibrosis, G551D-CFTR mutation. Top |