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
"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
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: http://www.cysticfibrosis.ca/en/Advocacy/Kalydeco.php
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 www.cysticfibrosis.ca.
SOURCE: Cystic Fibrosis Canada
For further information:
Melinda McInnes, Communications Director
Cystic Fibrosis Canada
Tel: 416-485-9149 ext. 240
Toll free: 1-800-378-2233 ext. 240