Rare Daily Staff

The European Commission approved Alnylam Pharmaceuticals’ Onpattro, a first-of-its-kind RNA interference therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy.

The decision follows the U.S. Food and Drug Administration’s approval of Onpattro earlier this month.

Hereditary transthyretin-mediated amyloidosis is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene 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, as well as other disease manifestations. The median survival is 4.7 years following diagnosis, with a reduced survival of 3.4 years for patients presenting with cardiomyopathy.

Onpattro is an intravenously administered RNAi therapeutic targeting transthyretin in development for the treatment of hATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to reduce the deposition and facilitate the clearance of TTR amyloid in peripheral tissues and potentially restore function to these tissues. It is the first and only FDA-approved treatment for this indication.

In clinical studies, Onpattro was shown to improve polyneuropathy – with reversal of neuropathy impairment in a majority of patients – and to improve a composite quality of life measure, reduce autonomic symptoms, and improve activities of daily living.

“Until recently, patients diagnosed with hereditary ATTR amyloidosis faced an uncertain future. A lack of treatments to halt or reverse the progression of disease resulted in a gradual and inescapable decline in their day-to-day functioning, placing a heavy burden not just on the patient themselves but on their partners and families, many of whom end up being full-time carers,” said Philip Hawkins, of the National Amyloidosis Centre, Division of Medicine, and University College London Medical School, Royal Free Hospital, UK. “Patisiran has the potential not only to transform these patients’ lives but to change the way in which we think about and treat hereditary ATTR amyloidosis.”

August 30, 2018

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