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Onpattro, Tegsedi Cardiac Imaging And Serum Biomarker Data Failed To Impress US FDA

 
• By Sue Sutter

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.

Drug Review Profile regular column
For both Onpattro and Tegsedi, FDA reviewers recommended limiting the indications to hATTR patients with polyneuropathy.

Imaging and serum marker data were not enough to persuade the US Food and Drug Administration about the purported cardiac benefits of the first two drugs approved for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) patients.

The agency did not put much stock in the exploratory data on cardiac benefits provided by Alnylam Pharmaceuticals Inc....

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