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...
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