KALYDECO® (ivacaftor) is a prescription medicine for the treatment of cystic fibrosis in patients ages six and older who have the specific G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or G970R mutation, or ages 18 and older who have the R117H mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. In Canada, the number of patients with these mutations is about 180. 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 for patients with the G551D mutation. Approval for the additional 9 mutations was received on June 6, 2014, and approval for R117H was received on March 13, 2015.
On March 26, 2013, it was announced that the Common Drug Review’s (CDR) Canadian Drug Expert Committee (CDEC) 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. Cystic Fibrosis Canada submitted patient input to CDEC for the additional mutations on July 22, 2014 and it received a positive recommendation on December 19, 2014.
Following the CDR recommendation for G551D, Kalydeco was subject to the Pan-Canadian Pharmaceutical Alliance (PCPA), where negotiations with Vertex were led by Alberta on behalf of the provinces (with the exception of Quebec). A letter of intent was entered into between the participating PCPA jurisdictions (Quebec excluded) on a pricing agreement for Kalydeco on June 16, 2014. A letter of intent typically sets out the standard terms for health ministries and Vertex when considering provincial coverage for Kalydeco. Unfortunately, the additional 9 gating mutation indications did not make it through the pCPA negotiations.
For patients with private insurance who have been prescribed Kalydeco by their healthcare provider for one of the 10 mutations indicated, kalydeCARE™ is a Patient Assistance Program which can help navigate the reimbursement system. For more information please call: 1-855-CARE-571 (1-855-227-3571).
Cystic Fibrosis Canada remains committed to advocating for access to Kalydeco for Canadians with CF who have the remaining mutationsWe want to thank all our advocates especially those patients and families personally impacted by Kalydeco for their tenacity and courage in the fight for Kalydeco.