Alnylam Introduced First and Only AMVUTTRA® (vutrisiran) for ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)
Alnylam Pharmaceuticals has AMVUTTRA® (vutrisiran) as the first and only FDA-approved treatment for adults with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM) to reduce cardiovascular mortality, hospitalisations, and urgent heart failure visits.
ATTR-CM is a progressive condition caused by the accumulation of misfolded transthyretin (TTR) proteins, leading to severe heart damage and significantly reducing life expectancy. It affects an estimated 150,000 people in the United States and over 300,000 globally.
Many patients remain undiagnosed or experience disease progression despite existing treatments.
Approved therapy utilises RNAi technology to reduce the production of TTR, addressing the underlying cause of ATTR-CM. It is administered via subcutaneous injection every three months, preventing the accumulation of harmful amyloid deposits in the heart.
Approval was based on findings from the HELIOS-B Phase 3 clinical trial, which assessed the safety and effectiveness of AMVUTTRA in ATTR-CM patients. Results showed a significant reduction in cardiovascular-related deaths and hospitalisations compared to placebo.
This groundbreaking approval indication of AMVUTTRA, making it the first FDA-approved therapy for both ATTR-CM and hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTRN).
