Calling for a timely approach to make PrORKAMBITM accessible and affordable for Canadians with the most common CF gene mutation
Health Canada has issued a Notice of Compliance for the lumacaftor/ivacaftor combination drug PrORKAMBITM for use in Canada. This gives Canadians with cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation a chance to gain access to this breakthrough treatment. PrORKAMBITM could benefit around 1,550 Canadians living with CF.
"Like Kalydeco before it, PrORKAMBITM treats the underlying defect in cystic fibrosis, but for a much larger part of the CF population. Health Canada’s approval of PrORKAMBITM is the first step in getting this new medicine into the hands of the people who need it most. We congratulate Vertex on the approval of PrORKAMBITM by Health Canada and look forward to working with Vertex and the provincial drug plans to ensure access to PrORKAMBITM is made available to eligible CF patients across Canada,” says John Wallenburg, Cystic Fibrosis Canada’s Chief Scientific Officer.
PrORKAMBITM improves lung function and significantly reduces the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and accelerated lung disease. It is the second drug approved by Health Canada that targets the underlying cause of CF. Ivacaftor (as a single therapy) was the first. For a CF patient with two copies of the F508del mutation access to PrORKAMBITM could lead to a healthier, longer life.
“Among my many other roles, I am a wife, a mother, and a healthcare professional. I am also a woman living and sometimes struggling with cystic fibrosis. Gaining access to PrORKAMBITM gives me hope that I will have the opportunity to grow old with my husband, watch my children mature into adults, and choose when I am ready to retire because I have had a full and satisfying career, and not because my illness demanded it,” says Megan Parker.
Vertex, the manufacturer of PrORKAMBITM has submitted the drug to the Common Drug Review (CDR), a process run by the Canadian Agency for Drugs and Technologies in Health (CADTH), Quebec has its own process. A team of independent experts will review PrORKAMBITM 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, all provincial health ministries will review PrORKAMBI™ for reimbursement by publicly funded drug plans.
Cystic Fibrosis Canada is calling for a compassionate and timely approach from the drug manufacturer and drug plan administrators when it comes to making PrORKAMBITM accessible to the Canadians with CF that would benefit from this life-changing treatment.
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 also builds 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 more than $226 million in leading research, care and advocacy, resulting in one of the world’s highest median survival rates for Canadians living with cystic fibrosis. For more information, visit www.cysticfibrosis.ca.
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For more information or to set up interviews with spokespeople, please contact:
Andrea Smith, Senior Associate Marketing and Communications
Cystic Fibrosis Canada
Tel: 416-485-9149 ext. 291
Tel: 1-800-378-2233 ext. 291
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