Research Article Ivacaftor Therapy in CF Patients: Single Center Experience Pritish Mondal, 1 Amber Loyson, 1 Jorge Lascano, 2 and Satyanarayan Hegde 3 1 Department of Pediatrics, University of Florida, 1600 SW Archer Road, Suite HD 604, P.O. Box 100296, Gainesville, FL 32610, USA 2 Division of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, University of Florida, 1600 SW Archer Road, P.O. Box 100225, Gainesville, FL 32610, USA 3 Division of Pediatric Pulmonary Medicine, Department of Pediatrics, University of Florida, 1600 SW Archer Road, P.O. Box 100296, Gainesville, FL 32610, USA Correspondence should be addressed to Satyanarayan Hegde; hegdes@ul.edu Received 8 May 2014; Revised 3 September 2014; Accepted 15 September 2014; Published 22 October 2014 Academic Editor: Gernot Zissel Copyright © 2014 Pritish Mondal et al. his is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Ivacator is the irst novel cystic ibrosis pharmaceutical that acts at the molecular level to potentiate cystic ibrosis transmembrane conductance regulator (CFTR) function and was irst approved for clinical use in 2012. We are sharing our single center experience of ive patients: four from pediatric age group and one adult patient. All patients had both subjective and objective improvements in their health. Despite established lung disease, our patients had signiicant improvement in both their FEV1 (forced expiratory volume in 1 second) and FEF 25–75 and BMI (body mass index). Larger studies demonstrated only 6.7% improvement in mean FEV1 ater starting Ivacator therapy but their patient population had normal lung function to begin with. In contrast our case series demonstrates that, in patients with established lung disease and diminished lung function, Ivacator can be expected to result in much higher recovery in lung function. Mean FEV1 improved by 35% in our case series. Ivacator is extremely expensive, costing $300,000 per patient per year requiring lifelong therapy, hence requiring prior authorizations from most third-party payers in the USA. he knowledge shared from our experience will be useful for other clinicians to petition healthcare policymakers on behalf of their patients. 1. Introduction Cystic ibrosis (CF) is the most common life-threatening autosomal recessive disease in the USA [1]. here is a wide variety of gene sequences in cystic ibrosis and more than 1900 CF mutations have been identiied [2]. Although daily airway clearance therapies and pancreatic enzyme replacement are keys to CF management, the introduction of Pulmozyme and the introduction of inhaled antibiotics were two milestones in CF management and have become routine care in established lung disease in CF patients. However, there was no known medication that could correct this disease and its underlying molecular defect until recently. Ivacator, also known as Kalydeco, is the irst marketed drug which restores the function of the cystic ibrosis transmembrane conductance regulator (CFTR). It was approved by the United States Food and Drug Administration (FDA) in January 2012 for treating cystic ibrosis patients with G551D mutation, older than six years. It has shown to be efective as evidenced by reduction in the sweat chloride content of the subjects as well as by the improvement in FEV1 up to 10 percent [3]. Due to its prohibitive cost (approximately $300,000/ patient/year), Ivacator therapy requires prior authoriza- tion from insurers. hese insurers oten rely on published postmarketing data in order to add newer therapies to their approved lists. In premarketing clinical trials, Ivacator demonstrated sustained improvement in FEV1, body mass index (BMI) and the frequency of pulmonary exacerbations [3]. Recently Rowe et al. [4] published a large prospec- tive study with six-month followup of Ivacator therapy demonstrating modest but signiicant improvement in lung function in relatively healthy patients. We are sharing our single center experience of ive patients at the University of Florida Pediatric and Adult Cystic Fibrosis Program. We have followed up our patients for 15 months. Hindawi Publishing Corporation Advances in Medicine Volume 2014, Article ID 947923, 5 pages http://dx.doi.org/10.1155/2014/947923