Cystic Fibrosis Canada Calls on Premiers to Expedite Access to Expensive Drug

TORONTO, July 24, 2013 /CNW/ - As Premiers gather at the summer meeting of the Council of the Federation in Niagara-on-the-Lake, Cystic Fibrosis Canada calls for compassionate and quick action in providing about 100 cystic fibrosis (CF) patients with access to KALYDECO™, a new novel therapy that treats the underlying cause of cystic fibrosis for a small segment of people living with this fatal disease.

"As a mother of two adult children with cystic fibrosis, I know first-hand how precious every minute is for people battling this disease," said Debra Berlet, Chair of Cystic Fibrosis Canada Board of Directors. "In Canada, about 20 patients are fortunate to have access to Kalydeco thanks to their private insurance. This has now become an issue of equity, as those without the benefit of a generous health insurance plan continue to be denied this much needed drug."

Kalydeco was approved by Health Canada for use by CF patients aged six years and older who have the specific G551D mutation gene in November 2012. This drug received a positive recommendation with conditions from the Canadian Drug Expert Committee in March 2013. The conditions include a substantial reduction in price in order for the drug to be considered cost-effective and development of clinical criteria for the discontinuation of treatment in patients who fail to demonstrate a meaningful response. Kalydeco is now subject to the pan-Canadian pricing alliance process established by the Council of the Federation in 2010.

"I was diagnosed with cystic fibrosis at seven months and today my lung function has been reduced to about 40 per cent of a healthy person and my body weight is at 58 kilograms," said Tim Vallillee, a 45-year-old Nova Scotia man with the G551D CF gene mutation. "As soon as I get ill, it goes straight to my chest. My biggest fear is getting pneumonia, which can be deadly."

This drug helps to improve the function of the defective protein, leading to better lung function, weight gain and lower sweat chloride levels. For a CF patient with the specific G551D mutation, access to Kalydeco could lead to a healthier, longer life.

"Kalydeco is a breakthrough medication, one that is capable of reversing the basic defects of a very specific genetic mutation among a subpopulation of CF patients," said Dr. Harvey Rabin, Professor of Medicine and Microbiology and Infectious Diseases at the University of Calgary and Director of the CF Clinic at Calgary's Foothills Medical Centre. "It is proving to reverse the decline of pulmonary function and promote weight gain while preventing pulmonary infection exacerbations. Kalydeco is a great example of the future of personalized medicine."

Cystic Fibrosis Canada is appealing to Canada's Premiers to make Kalydeco available to those patients across the country who need it.

Background: Access to KALYDECO™ in Canada
Of the 4,000 Canadians living with this devastating disease, about 100 CF patients in Canada have the specific G551D mutation of the CFTR gene that can be treated by Kalydeco. (Source: Canadian CF Registry). When taken twice a day with fat-containing food, Kalydeco helps the protein made by the CFTR gene function better, improving lung function and weight gain. (Source: FDA)

Kalydeco has been available to patients in the United States through private insurance or state Medicaid plans since 2012. In 2013, England, Scotland, Wales and Northern Ireland and the Republic of Ireland started funding Kalydeco for use by their patients. Kalydeco is being reviewed in Australia for inclusion in the national Pharmaceutical Benefits Scheme.

In April 2013, Alberta Health has taken the lead on behalf of provinces in negotiations with Vertex Pharmaceuticals as part of the pan-Canadian pricing alliance process. Quebec is not part of this initiative.

For more information on KALYDECOTM, visit our dedicated page:

Cystic Fibrosis
Cystic fibrosis is the most common fatal genetic disease affecting Canadian children and young adults. It is a multi-system disease that affects mainly the lungs and the digestive system. In the lungs, where the effects are most devastating, a build-up of thick mucus causes severe respiratory problems. Mucus and protein also build up in the digestive tract, making it difficult to digest and absorb nutrients from food. As improved therapies have helped to address the malnutrition issues, ultimately most deaths related to cystic fibrosis are due to lung disease. There is no cure.

Cystic Fibrosis Canada
Cystic Fibrosis Canada is one of the world's top three charitable organizations committed to finding a cure for cystic fibrosis and is an internationally-recognized leader in funding CF research, innovation, and clinical care. We invest more funding in life-saving CF research and care than any other non-governmental agency in Canada. Since 1960, Cystic Fibrosis Canada has invested almost $150 million in leading research, care and advocacy, resulting in one of the world's highest survival rates for Canadians living with cystic fibrosis. For more information, please visit

SOURCE: Cystic Fibrosis Canada

For further information:

Media Contact: 
Melinda McInnes, Communications Director
Cystic Fibrosis Canada
Tel: 416-485-9149 ext. 240
Toll free: 1-800-378-2233 ext. 240

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