regulatory
confidence high
sentiment positive
materiality 0.90
Alnylam gets FDA approval for AMVUTTRA in ATTR-CM, first RNAi therapy for cardiomyopathy
ALNYLAM PHARMACEUTICALS, INC.
- FDA approved AMVUTTRA for ATTR-CM to reduce cardiovascular mortality, hospitalizations, and urgent heart failure visits.
- HELIOS-B trial showed 28% risk reduction in all-cause mortality + recurrent CV events; 36% mortality reduction through 42 months overall.
- First and only FDA-approved therapy covering both ATTR-CM and hATTR-PN manifestations in adults.
- About 150K US patients affected; Alnylam expects $0 out-of-pocket cost for majority under Alnylam Assist program.
- Marketing applications under review by EMA, ANVISA, PMDA; further global submissions planned in 2025.