inotersen

Inotersen (Tegsedi) is an antisense oligonucleotide (ASO) therapeutic developed by Ionis Pharmaceuticals and its subsidiary Akcea Therapeutics for the treatment of polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR). It is a 2'-O-methoxyethyl modified phosphorothioate oligonucleotide that specifically binds to the messenger RNA (mRNA) of the transthyretin (TTR) gene. This binding induces the degradation of both mutant and wild-type TTR mRNA by RNase H1, leading to a significant reduction in the synthesis and circulating levels of TTR protein. By lowering TTR levels, inotersen prevents the formation and deposition of amyloid fibrils in peripheral nerves and other tissues. Approved by the FDA in 2018, it was administered as a weekly subcutaneous injection. However, as of late 2024, it has been discontinued in the United States market for commercial reasons, though it remains available in other regions.

Brand names

Tegsedi

Other names

inotersen sodium