AMVUTTRA
AMVUTTRA (vutrisiran) is a quarterly subcutaneous RNAi therapy developed by Alnylam that silences the TTR gene in liver cells, preventing production of the transthyretin protein that misfolds and deposits as amyloid in ATTR amyloidosis. In ATTR polyneuropathy, amyloid damages peripheral nerves; in ATTR cardiomyopathy, deposits in the heart muscle cause progressive heart failure. AMVUTTRA is approved for hereditary ATTR polyneuropathy and demonstrated a 28% relative reduction in a composite cardiovascular outcomes endpoint in Phase 3, supporting a pending cardiomyopathy label expansion with a December 2026 PDUFA.
Upcoming catalysts
Programs
ATTR Cardiomyopathy
ATTR cardiomyopathy is a progressive fatal heart disease caused by transthyretin amyloid deposits in the heart muscle — increasingly recognized as a significant cause of heart failure in older adults — with current approved treatments being TTR stabilizers. AMVUTTRA's Phase 3 HELIOS-B trial demonstrated a 28% relative reduction in a composite cardiovascular endpoint over 36 months, supporting a December 2026 PDUFA for label expansion into ATTR-CM alongside Pfizer's tafamidis and BridgeBio's acoramidis.