Inotersen

Generic Name
Inotersen
Brand Names
Tegsedi
Drug Type
Biotech
Chemical Formula
-
CAS Number
1492984-65-2
Unique Ingredient Identifier
0IEO0F56LV
Background

Inotersen is a transthyretin-directed antisense oligonucleotide for the treatment of the polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adults. It was FDA approved in October 2018. Inotersen has been shown to improve the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis .

Hereditary transthyretin amyloidosis is caused by single-nucleotide variants in the gene encoding transthyretin (TTR), which lead to transthyretin misfolding and the deposition of amyloid substance systemically. Progressive amyloid accumulation may lead to multiorgan dysfunction and death .

Indication

Inotersen is a transthyretin-directed antisense oligonucleotide indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Associated Conditions
Hereditary Transthyretin Amyloidosis, Polyneuropathies caused by Hereditary transthyretin-mediated amyloidosis
Associated Therapies
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