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Review
. 2020 Dec;9(2):301-315.
doi: 10.1007/s40120-020-00208-1. Epub 2020 Aug 12.

A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis

Ivan Urits  1 , Daniel Swanson  2 , Michael C Swett  2 , Anjana Patel  2 , Kevin Berardino  2 , Ariunzaya Amgalan  2 , Amnon A Berger  3 , Hisham Kassem  4 , Alan D Kaye  5 , Omar Viswanath  6   7   8
Affiliations
Review

A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis

Ivan Urits et al. Neurol Ther. 2020 Dec.

Erratum in

Abstract

Hereditary variant transthyretin amyloidosis (ATTRv) is a rare genetic defect that affects about 5000-10,000 people worldwide, causing amyloidosis secondary to misfolding of mutant transthyretin (TTR) protein fibrils. TTR mutations can cause protein deposits in many extracellular regions of organs, but those deposits in cardiac and axonal cells are the primary cause of this clinical syndrome. Treatment options are limited, but new drugs are being developed. Patisiran, a novel drug, is a liposomal siRNA against TTR that specifically targets this protein, reducing the accumulation of TTR in tissues, with subsequent improvement in both neuropathy and cardiac function. Patisiran is likely to serve as a prototype for the development of further intelligent drug solutions for use in targeted therapy. In this review we summarize the evidence currently available on the treatment of polyneuropathy in people with ATTRv with patisiran. We review the evidence on its efficacy, safety, and indications of use, citing novel and seminal papers on these subjects.

Keywords: Cardiac amyloidosis; Hereditary amyloidosis; Orphan drugs; Polyneuropathy.

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Conflict of interest statement

All authors (I Urits, D Swanson, MC Swett, A Patel, K Berardino, A Amgalan, AA Berger, H Kassem, AD Kaye, O Viswanath) have nothing to disclose.

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