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Gales L. Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis. Pharmaceuticals (Basel). 2019;12(2)doi: 10.3390/ph12020078.
Gales, L. (2019). Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis. Pharmaceuticals (Basel, Switzerland), 12(2), . https://doi.org/10.3390/ph12020078
Gales, Luís. "Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis." Pharmaceuticals (Basel, Switzerland) vol. 12,2 (2019). doi: https://doi.org/10.3390/ph12020078
Gales L. Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis. Pharmaceuticals (Basel). 2019 May 21;12(2). doi: 10.3390/ph12020078. PMID: 31117178; PMCID: PMC6631675.
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Pharmaceuticals (Basel). 2019 May 21;12(2). doi: 10.3390/ph12020078.

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

Pharmaceuticals (Basel, Switzerland)

Luís Gales

Affiliations

  1. i3S-Instituto de Investigação e Inovação em Saúde, Rua Alfredo Allen, 208 Porto, Portugal. [email protected].
  2. IBMC-Instituto de Biologia Molecular e Celular Universidade do Porto, Rua Alfredo Allen, 208 Porto, Portugal. [email protected].
  3. ICBAS-Instituto de Ciências Biomédicas Abel Salazar, Rua de Jorge Viterbo Ferreira 228 Porto, Portugal. [email protected].

PMID: 31117178 PMCID: PMC6631675 DOI: 10.3390/ph12020078

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.

Keywords: Tegsedi (Inotersen); antisense oligonucleotide; gene silencing; hATTR treatment; hereditary transthyretin-mediated amyloidosis (hATTR); transthyretin

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