KALYDECO® (ivacaftor) is a prescription medicine for the treatment of cystic fibrosis in patients aged:
- Twelve months and older and weighing 7 kg to less than 25 kg who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
- Six and older and weighing 25 kg or more who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
- Eighteen years and older with an R117H mutation in the CFTR gene.
In Canada, the number of patients with these mutations is over 200. 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 June 12, 2020 the pan-Canadian Pharmaceutical Alliance (pCPA) agreed to enter negotiations with Vertex Pharmaceuticals on Orkambi and Kalydeco. Cystic Fibrosis Canada is calling on governments and Vertex to get through these negotiations quickly and to work together to fast-track access to Trikafta. For more information please see the Q&A.
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 Canada’s Common Drug Review 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 the manufacturer – 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 gating mutation indications did not make it through the pCPA negotiations at that time.
CF Canada has continued to push for the remaining mutations to be covered. In the summer of 2019 – five years after the addition of the remaining mutations - the pCPA and the manufacturer completed price negotiations on these mutations. CF Canada has encouraged governments to fast-track coverage of the remaining mutations.
For more information on access through provincial, territorial or federal coverage of Kalydeco, please speak to your CF clinical team. You may also wish to contact kalydeCARE™, 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 medicines for Canadians with CF. We want to thank all our advocates, especially those patients and families personally impacted by Kalydeco for their tenacity and courage in the fight for access.