Amyloidosis Drug PROs Supported Clinical Meaningfulness Of Objective Measures

18 Apr 2019

In pivotal trials for Alnylam’s Onpattro and Ionis’ Tegsedi, statistically persuasive results on a patient-reported, quality-of-life measure were needed to show the drugs had a meaningful benefit on how hATTR patients with polyneuropathy feel and function, US FDA said.

Alnylam Pharmaceuticals Inc.'s Onpattro (patisiran) and Ionis Pharmaceuticals Inc.’s Tegsedi (inotersen) – the first two treatments for hereditary transthyretin-mediated amyloidosis (hATTR) – secured US Food and Drug Administration approval on the strength of results from an objective efficacy assessment and a subjective quality-of-life measure the agency emphasized for its clinical meaningfulness.

For the pivotal trials of both drugs, the FDA urged the sponsors to incorporate the Norfolk Quality of Life-Diabetic Neuropathy...