Inotersen: First Global Approval

Ionis Pharmaceuticals and Akcea Therapeutics have developed inotersen (Tegsedi™), an antisense oligonucleotide inhibitor of mutant and wild-type human transthyretin (TTR), for the treatment of hereditary transthyretin amyloidosis (hATTR). Mutation of the TTR gene results in accumulation of TTR prote...

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Bibliographic Details
Published in:Drugs (New York, N.Y.) N.Y.), 2018-09, Vol.78 (13), p.1371-1376
Main Author: Keam, Susan J.
Format: Article
Language:English
Subjects:
AdisInsight Report
Amyloid Neuropathies, Familial - drug therapy
Amyloidosis
Antisense oligonucleotides
Cardiomyopathy
Conflicts of interest
Disease
Disease Progression
Drug Approval
Europe
Genotype & phenotype
Humans
Internal Medicine
Liver
Medicine
Medicine & Public Health
mRNA
Mutation
Nervous system
Oligonucleotides - therapeutic use
Oligonucleotides, Antisense - antagonists & inhibitors
Organs
Patients
Peripheral nervous system
Pharmaceuticals
Pharmacology/Toxicology
Pharmacotherapy
Polyneuropathies - drug therapy
Polyneuropathy
Prealbumin - metabolism
Protein biosynthesis
Proteins
Quality of Life
Transcription
Transthyretin
Treatment Outcome
Vitamin A
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Summary:Ionis Pharmaceuticals and Akcea Therapeutics have developed inotersen (Tegsedi™), an antisense oligonucleotide inhibitor of mutant and wild-type human transthyretin (TTR), for the treatment of hereditary transthyretin amyloidosis (hATTR). Mutation of the TTR gene results in accumulation of TTR protein fragments as amyloid deposits throughout the organs in patients with hATTR, including the peripheral nervous system and the heart. Treatment with inotersen, which selectively binds to TTR mRNA, prevents the synthesis of TTR protein in the liver, thus reducing further amyloid deposition throughout the body. Subcutaneous administration of inotersen significantly reduced neurological progression and improved health-related quality of life in patients with hATTR and polyneuropathy in a phase III trial. Based on these results, inotersen was recently approved in the EU for the treatment of stage 1 or 2 polyneuropathy in adult patients with hATTR and is under evaluation in the USA and Canada for a similar indication. This article summarizes the milestones in the development of inotersen leading to this first approval.
ISSN:0012-6667
1179-1950
DOI:10.1007/s40265-018-0968-5