An FDA advisory committee voted 9 to 3 that the benefits of patisiran outweigh the risks for ATTR cardiomyopathy, but many questioned whether the benefit is clinically meaningful.
Identification of an antibody linked to spontaneous reversal of cardiac transthyretin amyloidosis may represent a novel approach to treatment of this progressive and fatal condition.
The recombinant human antibody NI006 was safe for the treatment of transthyretin amyloid cardiomyopathy in a small phase 1 trial and may also reduce amyloid protein accumulation in the heart.
The investigational antisense agent eplontersen slows disease progression and im-proves quality of life in hereditary transthyretin-mediated amyloid polyneuropathy, final data from a phase 3 trial indicate.
The document provides a diagnostic algorithm and emphasizes the need for a multidisciplinary, individualized approach for this under-recognized cause of heart failure.