Onpattro (patisiran) has been approved in the US since 2018 as a treatment for polyneuropathy caused by ATTR, but Alnylam was hoping to expand its use to include patients with the disease who are experiencing cardiac complications of the disease.
ATTR is characterised by the formation of amyloid fibril deposits in tissues and organs, in the heart in the case of patients with cardiomyopathy, causing the left ventricle to become stiff and weak and raising the risk of heart failure.
