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Alnylam Ready To Expand TTR Franchise With Positive Cardiomyopathy Results

Supplemental Onpattro US Filing Planned For Late 2022

 
• By Mandy Jackson

ATTR amyloidosis patients with cardiomyopathy had statistically significant functional and quality of life improvements when treated with Onpattro (patisiran) in the Phase III APOLLO-B study.

RNA, Epigenetics concept, 3d illustration
Onpattro silences the RNA that makes the TTR protein • Source: Shutterstock

Alnylam Pharmaceuticals Inc. has the data it needs to file for supplemental approval in the US in late 2022 for Onpattro (patisiran) in the treatment of transthyretin-mediated (ATTR) amyloidosis patients with cardiomyopathy now that the Phase III APOLLO-B clinical trial has met it primary and key secondary endpoints. The data disclosed on 3 August also support the company’s plan to extend its transthyretin (TTR)-silencing RNAi therapeutic franchise into a bigger ATTR amyloidosis population.

Onpattro was approved to treat ATTR amyloidosis polyneuropathy in 2018 and Amvuttra (vutrisiran) – a subcutaneously administered follow-on to Alnylam’s original I.V. drug – was approved this year in the ATTR polyneuropathy indication

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