Shots:

• The approval is based on P-III NEURO-TTR study trial assessing Tegsedi vs PBO in 172 patients in ratio (2:1) with hATTR amyloidosis with symptoms of polyneuropathy
• The study resulted in effective results in measures of neuropathy and QoL while measuring with a Neuropathy Impairment Score +7 (mNIS+7) & Norfolk QOL-DN total score with 80% reduction in TTR
• Tegsedi (inotersen) is a qw SC RNA-targeting drug used for the reduction in the production of TTR protein and has also received EU and Health Canada approval for 1 or stage 2 polyneuropathy in adult patients with hATTR. In April- 2018 Ionis granted Akcea worldwide rights to commercialize Tegsedi

Ref: Ionis | Image: USA today