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
- -
Summary report on authorisation – Tegsedi®
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