Cystic Fibrosis Canada welcomes Health Canada's approval of KALYDECO™

Cystic Fibrosis Canada welcomes Health Canada's approval of KALYDECO™ 
Calling for the manufacturer and drug insurance plans to ensure CF patients 
receive equitable and affordable access to KALYDECO™ 
TORONTO, Dec. 3, 2012 /CNW/ - Health Canada has issued a Notice of Compliance 
(NOC) for KALYDECO™, approving the drug for use in Canada. This gives 
Canadian cystic fibrosis (CF) patients with the G551D mutation (ages six and 
older) a chance to gain access to this new, breakthrough treatment. Cystic 
Fibrosis Canada calls on Vertex Pharmaceuticals Inc. and drug plan 
administrators to work together to resolve the high price of KALYDECO™ so 
that CF patients can access this drug in an equitable and affordable way. 
"The approval of KALYDECO™ is just the first step in bringing this new, 
breakthrough therapy to CF patients," said Ken Chan, Vice President, Advocacy, 
Research and Healthcare. "As one of the most expensive drugs in the world, CF 
patients simply cannot afford to pay for this much-needed medicine. Cystic 
Fibrosis Canada is calling for a compassionate approach from manufacturers and 
drug plan administrators when it comes to making new, life-changing medicines 
accessible to CF patients." 
KALYDECO™ is the first therapy that targets the underlying cause of cystic 
fibrosis. 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. Canada now joins the United States and the 
European Union in making KALYDECO™ available for use for CF patients. The 
G551D mutation is present in roughly 100 Canadians with cystic fibrosis. 
"When the gene responsible for cystic fibrosis was discovered in Canada back 
in 1989, one could only imagine the moment when that discovery would lead to a 
drug like KALYDECO™, said Cystic Fibrosis Canada President and CEO Maureen 
Adamson. "This is the beginning of good news for Canadian CF patients, and I 
look forward to even better news when this drug will be affordable and 
accessible to the patients who will benefit from it." Cystic Fibrosis Canada 
helped fund the international team that discovered the CF gene under the 
leadership of Dr. Lap-Chee Tsui at the Hospital for Sick Children in Toronto. 
Key opinion leaders throughout the Canadian CF clinical community also 
strongly support giving patients access to this drug. 
"KALYDECO™ is an incredible leap forward in CF therapy," said Dr. Larry 
Lands, CF Clinic Director at Montreal Children's Hospital. "Even though 
KALYDECO™ as a stand-alone treatment will only help a limited segment of 
affected patients, it shows promise as a combination therapy for other 
patients with cystic fibrosis, and points the way towards the development of 
tailored treatments that will correct the underlying problem for each and 
every affected person with cystic fibrosis." 
"This treatment represents a paradigm shift in therapy; it's the first one to 
target the basic defect in the cells of CF patients," said Dr. Elizabeth 
Tullis, Head of Respirology and Director of the Adult Cystic Fibrosis Program 
at St. Michael's Hospital in Toronto. "While KALYDECO™ is currently 
applicable only to a small proportion of CF patients; it gives us hope that a 
similar approach can be developed for all people with cystic fibrosis." 
"The future of some babies diagnosed through CF newborn screening is going to 
be revolutionized by KALYDECO™," said Dr. Mark Chilvers, CF Clinic Director, 
BC Children's Hospital. "CF Clinicians now have access to a life-changing 
therapy that targets this specific genetic mutation." 
In October 2012, Cystic Fibrosis Canada made a submission to the Common Drug 
Review (CDR) process in an effort to convince provincial drug plans to provide 
Canadian CF patients with access to KALYDECO™. Testimonials submitted for 
affected patients who are currently being treated with KALYDECO™ were 
compelling. They report transitions from debilitating illness and exhaustion 
to having more energy, being able to finally exercise, being able to spend 
more time with their families, and an overall enhanced quality of life. Many 
patients strongly believe that using KALYDECO™ will allow them to avoid lung 
transplantation for end-stage lung disease. Cystic Fibrosis Canada is awaiting 
the CDR recommendation before deciding on next steps. 
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 Patient Data 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) 
The Common Drug Review (CDR) was established by the federal, provincial, and 
territorial government to provide publicly-funded drug plans (except Quebec) 
with high-quality drug reviews that will inform their decision making. 
The high price of KALYDECO™ has meant CF patients in certain jurisdictions 
face barriers to accessing the drug despite KALYDECO™ receiving regulatory 
approval. For example, in England where KALYDECO™ has been approved for use 
since July 2012, the drug still remains out of reach to CF patients because of 
its cost ― estimated at over £180,000 per patient per year. In the U.S., 
KALYDECO™ costs around $294,000 per patient per year. 
Vertex, the manufacturer of KALYDECO™, has submitted the drug to the CDR, a 
process run by the Canadian Agency for Drugs and Technologies in Health 
(CADTH). A team of independent experts will review KALYDECO™, and compare 
how well the drug works to current CF treatments, and whether the drug 
provides value for money. Based on their findings, a recommendation on whether 
or not drug plans should cover the drug will be issued. After the CDR 
recommendation has been made, provincial health ministries (except for Quebec, 
which has a separate process) will review KALYDECO™ for reimbursement by 
publicly funded drug plans. 
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. Currently, 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 more than $140 million in leading research and care, 
resulting in one of the world's highest survival rates for Canadians living 
with cystic fibrosis. For more information, visit 
Melinda McInnes, Cystic Fibrosis Canada Program Director, Public Relations and 
Advocacy Communications Tel: 416-485-9149 ext. 240 | 1-800-378-2233 ext. 240 | 
Cell:  416-371-5196 
SOURCE: Cystic Fibrosis Canada 
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CO: Cystic Fibrosis Canada
ST: Ontario
-0- Dec/03/2012 15:13 GMT
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