Ancaster, ON, March 2, 2020 — Hereditary Amyloidosis Canada (HAC) is joined by the Regroupement québécois des maladies orphelines (RQMO) in commending the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d’excellence en santé et services sociaux (INESSS) for both recommending that Tegsedi™ (inotersen) and Onpattro™ (patisiran) be reimbursed by public drug plans for the treatment of polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis (hATTR). 

“We’re so pleased that the voices of hATTR amyloidosis patients, families and their caregivers are being heard regarding the dire need for publicly funded access to new treatment options like Tegsedi and Onpattro,” said Anne Marie Carr, Founder and President of Hereditary Amyloidosis Canada. “We urge all provincial and territorial governments to make these therapies accessible through their respective drug plans as quickly as possible to allow for the best possible outcomes for patients living with this life-limiting disease.”

hATTR amyloidosis is a rare, genetic disease which can lead to significant disability and death. The disease occurs when there is a build-up of abnormal or “misfolded” proteins – known as amyloid fibrils – in the body’s tissues and/or organs. The progressive accumulation of amyloid deposits leads to sensory, motor and autonomic dysfunction, often having debilitating effects on multiple aspects of a patient’s life. Left untreated, the life expectancy of hATTR amyloidosis patients can be as little as 2.5 years from symptom onset. Until recently, the only available treatment for hATTR amyloidosis was liver transplant.

“Early and accurate diagnosis is important due to the rapid, natural progression of hATTR amyloidosis,” said Dr. Vera Bril, Professor of Medicine at the University of Toronto and Director of the Neuromuscular Section of Neurology at the University of Toronto. “The approval of new treatments specifically designed to prevent the formation of abnormal amyloid proteins in the body has created a new urgency to appropriately treat patients who may benefit from these new therapies.”

In October 2018, Health Canada approved Tegsedi as the first treatment for polyneuropathy in adult patients with hATTR amyloidosis, followed by the approval of a second treatment, Onpattro, in July 2019. The manufacturers of both treatments are currently in active negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA).

“Due to the complexity of this rare genetic disease, patients can experience a lengthy delay in diagnosis, making it even more important to ensure that all approved treatment options are accessible to patients no matter where they live in Canada,” said Gail Ouellette, a geneticist, genetic counsellor and Executive Director of Regroupement québécois des maladies orphelines (RQMO).

To learn more about access to hATTR amyloidosis treatments in Canada, or to get involved in HAC’s advocacy efforts around this issue, please visit: https://madhattr.ca/advocacy/.

About Hereditary Amyloidosis Canada (HAC)

Hereditary Amyloidosis Canada (HAC) is a patient advocacy organization founded by and for people who are personally impacted by this rare disease. The non-profit organization’s mission is to be a source of support and information, and to advocate for access to health services and treatments and other issues relevant to Canadian patients. For more information, visit www.madhattr.ca.

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For more information:

Anne Marie Carr                                  
Founder and President
Hereditary Amyloidosis Canada
info@madhattr.ca

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