Ivacaftor Case Study

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FDA approved Ivacaftor on January 31,2012. It is used to treat Cystic Fibrosis in 6 years or older. Ivacaftor is the first drug that treats an underlying cause of Cystic Fibrosis(CF), rather than the symptoms of the disease. CF is not curable, the treatment is focused on alleviation of symptoms and treatment of complications. Proposed Dosage: 150 mg twice daily (300 mg/day). Route of Administration: Oral Mechanism of action: Ivacaftor acts as a CFTR (Cystic Fibrosis Transmembrane Conductance Regulator). The CFTR protein is a chloride channel found in the surface of epithelial cells throughout the body. Mutation of the CFTR gene may result in a reduced number of CFTR channels at the cell surface, abnormal channel function, or both. Ivacaftor…show more content…
The absorption of ivacaftor in mouse, rats, rabbits and dogs was rapid through oral administration. The bioavailability ranged from 30-100% Distribution: Ivacaftor was rapidly distributed across all tissues in rats. It was highly distributed in the GI tract followed by liver and kidney. Metabolism: Ivacaftor produces M1 and M6 metabolites. Ivacaftor was extensively retained by the liver by CYP3A. M1 is pharmacologically active and retains 1/6th of the potency of the parent drug, whereas M6 is not pharmacologically active and retains 1/5th of the potency of the parent drug. M1 and M6 were proved to be disproportionate in humans. The appropriate dosage for the children will be 150mg/per day to retain the potency of the drug. Excretion: After taking Ivacaftor orally, the majority of ivacaftor was excreted in feces after metabolism of the drug. The metabolites M1 and M6 accounted for 22% and 43% respectively. There was no urinary excretion of Ivacaftor. The half life was 12 hours after a single dose. Efficacy studies: 2 randomized controlled trials have been identified investigating the safety and efficacy of…show more content…
Before clinical trials (human trials), this research is carried out to check the pharmacological, toxicological, pharmacokinetic activity of the drug using different species of animals. During this stage, safety data is collected and evaluated before carrying out human trials. It holds an importance place in drug development because it holds a baseline for the human trials. Based on non clinical trials NOAEL (No Observable Adverse Effect Levels) is determined before testing out the drug on patients. The non clinical development of Ivacaftor was satisfactory for the drug approval because it showed promised results when the drug was tested out on the animals. Since the discovery of CFTR gene mutation, there had to be a therapeutic agent that could act on the cell surface. FDA approval of Ivacaftor proved to be a beginning of new era as the drug is effective and could promise satisfactory results. Also it offers easy drug administration. This drug was considered as the wonder drug when it was approved in 2012. Thus, Ivacaftor holds an important place in the history of drug development due to its capability to treat the cause of the disease other than to treat the

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