Health Canada approves Onpattro to treat polyneuropathy in adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis

Alnylam Pharmaceuticals, Inc. the leading RNA interference (RNAi) therapeutics company, announced the Health Canada approval and immediate availability of Onpattro (patisiran) for the treatment of polyneuropathy in adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis .

Onpattro is the only treatment in Canada indicated for all stages of polyneuropathy associated with hATTR amyloidosis, a progressive, debilitating, chronic and often fatal disease. Onpattro is based on Nobel Prize-winning science and is the first ever RNAi therapeutic to be approved in Canada.

Transthyretin (TTR)-mediated amyloidosis (ATTR) is a hereditary, systemic disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier for thyroid hormones and retinol binding proteins. Mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy. In its severest form, ATTR represents a major unmet medical need with significant morbidity and mortality as an orphan disease.