Life Changing Treatment, Trikafta, is Advancing Through The Canadian Drug Approval Process
Innovative drug can treat up to 90% of Canadians Living with CF, with promise of transforming lives
Today the cystic fibrosis story is moving closer to transformational change, with access to a long-awaited, life-changing therapy now advancing through the Canadian drug approval process. The Canadian Agency for Drugs and Technologies in Health (CADTH), an important drug review body that evaluates the cost effectiveness of drugs, is now reviewing the cystic fibrosis (CF) drug Trikafta for age 12 plus for patients who have at least one F508del mutation. The fact that CADTH is moving forward with their review of Trikafta indicates that the manufacturer was granted an ‘aligned review,’ the fastest review route possible.
With an aligned review underway, what is typically a one or two year process could be shortened to less than a year. Cystic Fibrosis Canada awaits news that CADTH’s counterpart in Quebec, Institut national d'excellence en santé et services sociaux (INESSS), has also commenced their review of Trikafta.
This long awaited step follows more than a year of tireless advocacy from the Canadian cystic fibrosis community, clinicians, Cystic Fibrosis Canada and its partners together calling on the government and manufacturer to bring this innovative treatment to Canada.
Cystic fibrosis is the most common fatal genetic disease affecting Canadian children and young adults. Of the Canadians with cystic fibrosis who died in 2018, half were under the age of 33. Clinical trial data has proven that Trikafta is the greatest innovation in the history of cystic fibrosis treatments with a transformational impact on recipients which includes longer and healthier lives.
Trikafta was fast-tracked for access by the U.S Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Trikafta has received regulatory approval in 32 countries, 27 within the EMA centralized regulatory approval, in addition to receiving approval in the United States, United Kingdom, Norway, Iceland and Liechtenstein. Trikafta has received public reimbursement in the United States, United Kingdom, Ireland, Austria, Denmark, Germany, and Slovenia.
Canadian research released in August 2020 demonstrates that if Trikafta was brought to Canada quickly, as it has been in the United States, the United Kingdom and parts of Europe already, it could result in extraordinary health benefits by 2030, including 15% fewer deaths, 60% fewer people living with severe lung disease and an increased estimated median age of survival for a child born with cystic fibrosis of 9.2 years.
“We are thrilled to learn of CADTH’s review. While our community has waited far too long for access to this drug, the pace of review is promising. We encourage all decision-makers to move as quickly as possible to get this drug approved and reimbursed fully from coast to coast. Time is of the essence for us to save lives and transform the CF story” says Kelly Grover, President and CEO of Cystic Fibrosis Canada.
Cystic Fibrosis Canada continues to call on the pan-Canadian Pharmaceutical Alliance (pCPA) to include Trikafta in its current negotiations with Vertex on Orkambi and Kalydeco so that the process can be expedited.
The current review applies to people aged 12 and older. To date, the clinical trials that have been completed for Trikafta are for those 12 years of age and older, though trials are underway for those who are 6-11 years old.
About Cystic Fibrosis
Cystic fibrosis is the most common fatal genetic disease affecting Canadian children and young adults. There is no cure. Of the Canadians with cystic fibrosis who died in 2018, half were under the age of 33. Cystic fibrosis is a progressive, degenerative multi-system disease that affects mainly the lungs and 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. In addition to the physical effects of the disease, mental health concerns are emerging; anxiety and depression are common among this population. Double lung transplants are the final option for patients with end-stage disease; most fatalities of people with CF are due to lung disease.
Cystic Fibrosis Canada
Cystic Fibrosis Canada has dramatically changed the cystic fibrosis story. We have advanced research and care that has more than doubled life expectancy. Since being founded by parents in 1960, Cystic Fibrosis Canada has grown into a leading organization with a central role engaging people living with cystic fibrosis, parents and caregivers, volunteers, researchers and healthcare professionals, government and donors. We work together to change lives through treatments, research, information and support. Despite our remarkable progress together, we are not yet done. Not when a child with cystic fibrosis still has only a 50% chance of living to 52. We will keep pushing, keep going further until all people with cystic fibrosis can and do experience everything life has to offer — and enjoy everything life has to offer. Learn more at www.cysticfibrosis.ca
For more information, please contact:
Aisha Champness, Director
Tel: 647-218-1255
Email: achampness@cysticfibrosis.ca
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