Cystic Fibrosis Canada Welcomes Common Drug Review Recommendation for KALYDECO™

Calling on provincial governments to ensure CF patients receive equitable access to KALYDECO™

TORONTO, March 26, 2013 /CNW/ - As part of the Common Drug Review process, the Canadian Drug Expert Committee (CDEC) has recommended that the drug KALYDECO™ (ivacaftor) be listed on the formulary listing of publicly funded drug plans for the treatment of cystic fibrosis in patients age six years and older who have the G551D mutation in the Cystic Fibrosis Transmembrane conductance Regulator.

"On behalf of cystic fibrosis patients and their families, we welcome the recommendation of the Canadian Drug Expert Committee," said Maureen Adamson, President and CEO of Cystic Fibrosis Canada. "We are grateful for the opportunity to make a patient input submission as part of the Common Drug Review process and are pleased that the voices of our patients and their families were listened to. I look forward to even better news when this drug will be accessible to the patients who will benefit from it."

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. The G551D mutation is present in roughly 100 Canadians with cystic fibrosis.

"While this announcement is positive news, our efforts will now focus on asking provincial governments across the country to fund this drug as soon as possible," said Ken Chan, Vice President of Advocacy, Research and Healthcare at Cystic Fibrosis Canada. "We will be calling on provincial health ministers to join their counterparts in the United States, United Kingdom and Ireland in providing access to this breakthrough drug."

In October 2012, Cystic Fibrosis Canada made a submission to the Common Drug Review (CDR) process on the drug 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.

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)

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.

KALYDECO™ has been available to patients in the United States through private insurance or state Medicaid plans since 2012. In 2013, England, Scotland, Ireland and Northern Ireland started funding KALYDECO™ for use by their patients.

For more information on KALYDECO^TM, visit our dedicated page:

http://www.cysticfibrosis.ca/en/Advocacy/Kalydeco.php

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 www.cysticfibrosis.ca.

SOURCE: Cystic Fibrosis Canada