Ivacaftor (Kalydeco) 150 mg tablet for treatment of cystic fibrosis with G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or G970R mutation
The drug works by prolonging the time that activated CFTR channels remain open, thereby enhancing the regulation of chloride and water transport across cell membranes. It is available as a 150 mg oral tablet. The Health Canada recommended dose is 150 mg every 12 hours with fat-containing food. The m...
| Corporate Author: | |
|---|---|
| Format: | eBook |
| Language: | English |
| Published: |
Ottawa (ON)
Canadian Agency for Drugs and Technologies in Health
2015 Jul, 2015
|
| Series: | Common drug review
|
| Subjects: | |
| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | The drug works by prolonging the time that activated CFTR channels remain open, thereby enhancing the regulation of chloride and water transport across cell membranes. It is available as a 150 mg oral tablet. The Health Canada recommended dose is 150 mg every 12 hours with fat-containing food. The manufacturer is seeking a listing recommendation based on the Health Canada indication. Accordingly, a systematic review was undertaken to evaluate the beneficial and harmful effects of ivacaftor 150 mg for the treatment of CF in patients age six years and older who have a G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or G970R mutation in the CFTR gene Cystic fibrosis (CF), an autosomal recessive condition, is the most common fatal genetic disease affecting children and young adults in Canada. It is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, located on chromosome seven. This gene encodes for a chloride channel that regulates transport of salt and water across cell membranes. When CFTR is dysfunctional, secretions become tenacious and sticky, resulting in pathology in multiple organ systems, most notably the lungs and gastrointestinal tract. Although there is no cure for the underlying disease process, current therapies have increased the overall survival of CF patients, with the median life expectancy now at 48 years based on recent Canadian statistics. The goals of CF therapy until now have been: (1) preservation of lung function by minimizing pulmonary infection and inflammation; (2) restoration of baseline pulmonary function, symptoms, and level of inflammation following acute respiratory exacerbations; and (3) maintenance of adequate nutrition. Therapeutic strategy consists of a combination of physiotherapy, pharmacologic drugs (i.e., antibiotics, anti-inflammatory drugs, mucolytic drugs), nutritional treatments (i.e., high-calorie and high-fat diets) and pancreatic enzyme replacement for those with pancreatic insufficiency. To date, no therapies have addressed the underlying genetic defect or corrected the abnormal functioning of CFTR. Ivacaftor is a first-in-class oral CFTR potentiator approved by Health Canada for the treatment of CF in patients aged six years and older who have G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or G970R mutation in the CFTR gene. |
|---|---|
| Physical Description: | 1 online resource illustrations |