Patisiran

CHICAGO — RNA therapeutics improved survival, 6-minute walk distance and quality of life for patients with transthyretin amyloid cardiomyopathy, according to study results presented at the American College of Cardiology Scientific Session.

The FDA rejected a supplemental new drug application for patisiran to treat cardiomyopathy of wild-type transthyretin-mediated or hereditary transthyretin-mediated amyloidosis, citing insufficient evidence of clinical meaningfulness.

An FDA panel voted 9-3 in favor of expanding approval of patisiran to patients with cardiomyopathy of wild-type transthyretin-mediated or hereditary transthyretin-mediated amyloidosis, noting modest benefits but no significant safety concerns.

Evaluating efficacy and safety of patisiran (Onpattro, Alnylam) in adults with transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

In patients with transthyretin-mediated amyloidosis with cardiomyopathy, patisiran improved functional capacity and quality of life at 12 months compared with placebo, according to the results of the APOLLO-B trial.

Alnylam announced top-line results of a trial showing that patisiran improved 6-minute walk test distance in patients with transthyretin-mediated amyloidosis and cardiomyopathy.

Amyloidosis is a disease in which specific precursor proteins misfold into beta-pleated sheets. Misfolded proteins adhere to form oligomers resulting in insoluble amyloid fibrils that deposit into tissue.