FDA OKs Inotersen (Tegsedi) for Hereditary ATTR With Polyneuropathy

The US Food and Drug Administration (FDA) has approved inotersen (Tegsedi, Akcea Therapeutics and Ionis Pharmaceuticals) for the treatment of polyneuropathy (PN) in adults with hereditary transthyretin amyloidosis (hATTR).

hATTR-PN is a rare, progressive condition in which a mutation in the transthyretin (TTR) gene can lead to TTR amyloidosis, a severe, progressive deposition of amyloid in organs that results in multiorgan failure. Inotersen is an antisense oligonucleotide inhibitor of both mutant and wild-type TTR protein.

The FDA’s approval of inotersen was based on results from the phase 3 NEURO-TTR study, which were presented at the American Neurological Association Annual Meeting in 2017 and were reported by Medscape Medical News at that time.