Alnylam's Onpattro Carries Narrower hATTR Indication Without Cardiac Data
Executive Summary
US FDA still appears to be open, if not optimistic, about path towards broader label for patisiran, the first-of-its-kind targeted RNA-based therapy.
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Pivotal studies of the first two drugs approved for treating hereditary transthyretin-mediated amyloidosis did not provide cardiac efficacy data on how patients feel, function or survive, reviewers said; the agency approved Alnylam’s patisiran and Ionis’ inotersen for patients with the polyneuropathy form of the disease but not the cardiomyopathy form.