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.