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

• Published in: Neurology and therapy Vol. 9; no. 2; pp. 301 - 315
• Main Authors: Urits, Ivan, Swanson, Daniel, Swett, Michael C., Patel, Anjana, Berardino, Kevin, Amgalan, Ariunzaya, Berger, Amnon A., Kassem, Hisham, Kaye, Alan D., Viswanath, Omar
• Format: Journal Article
• Language: English
• Published: Cheshire Springer Healthcare 01.12.2020; Adis, Springer Healthcare
• Subjects: Cardiac amyloidosis; Hereditary amyloidosis; Internal Medicine; Medicine; Medicine & Public Health; Neurology; Orphan drugs; Polyneuropathy; Review; Orphan drugs; Cardiac amyloidosis; Polyneuropathy; Hereditary amyloidosis
• ISSN: 2193-8253; 2193-6536
• DOI: 10.1007/s40120-020-00208-1

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.