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Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis

1,2,3
1
i3S—Instituto de Investigação e Inovação em Saúde, Rua Alfredo Allen, 208 Porto, Portugal
2
IBMC—Instituto de Biologia Molecular e Celular Universidade do Porto, Rua Alfredo Allen, 208 Porto, Portugal
3
ICBAS—Instituto de Ciências Biomédicas Abel Salazar, Rua de Jorge Viterbo Ferreira 228 Porto, Portugal
Pharmaceuticals 2019, 12(2), 78; https://doi.org/10.3390/ph12020078
Received: 16 April 2019 / Revised: 16 May 2019 / Accepted: 17 May 2019 / Published: 21 May 2019
(This article belongs to the Special Issue The Story of Successful Drugs and Recent FDA-Approved Molecules)
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Abstract

Tegsedi (Inotersen) is a chemically modified antisense oligonucleotide that inhibits the hepatic production of transthyretin (TTR). Several single-point mutations in TTR destabilize its structure, leading to the aggregation and accumulation of amyloid deposits in the nervous system, heart, kidneys and eyes. In July 2018, Tegsedi was approved by the European Commission for use in adults with stage one and two polyneuropathies. Later on, in October 2018, the FDA and Health Canada also approved its use for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults in the U.S. and Canada. Tegsedi was developed by Ionis Pharmaceuticals, the company that holds the global marketing license, together with its subsidiary Akcea Therapeutics. View Full-Text
Keywords: Tegsedi (Inotersen); gene silencing; antisense oligonucleotide; transthyretin; hereditary transthyretin-mediated amyloidosis (hATTR); hATTR treatment Tegsedi (Inotersen); gene silencing; antisense oligonucleotide; transthyretin; hereditary transthyretin-mediated amyloidosis (hATTR); hATTR treatment
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Gales, L. Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis. Pharmaceuticals 2019, 12, 78.

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