Archive for the ‘ Cystic Fibrosis ’ Category

Ten thousand people suffering from cystic fibrosis have been offered new hope after a gene therapy was shown to reverse the condition in some cases.

The therapy, which was described by health experts as ‘life-changing’ is likely to be available within the next five years following a landmark trial.

cfclogo-enKALYDECO® (ivacaftor) is a prescription medicine for the treatment of cystic fibrosis in patients ages six and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. In Canada, the number of patients with this mutation is about 100. Kalydeco™, a pill taken twice a day with fat-containing food, helps the protein made by the CFTR gene function better and, as a result, improves lung function and other aspects of cystic fibrosis such as increasing weight.

On November 26, 2012, Health Canada approved Kalydeco™ for use in Canada. On March 26, 2012, it was announced that the Canadian Drug Expert Committee recommends that Kalydeco™ be listed on the formulary listing of publicly funded drug plans for the treatment of cystic fibrosis in patients age six and older who have the G551D mutation in the CFTR. Today, only a handful of CF patients in Canada who need Kalydeco™ have access through private insurance. Quebec has its own drug review process through the Institut national d’excellence en santé et en services sociaux (INESSS).



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