Alnylam Pharmaceuticals just announced that the FDA has accepted for review supplemental New Drug Application (sNDA) for vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). Vutrisiran is the generic name for AMVUTTRA®, which the FDA currently approves for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. By rapidly knocking down both mutant and wild‑type transthyretin (TTR), vutrisiran addresses the underlying cause of transthyretin amyloidosis (ATTR). If approved, Vutrisiran would become the first therapeutic approved in the U.S. to treat both the polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis. Congratulations to the Alnylam Pharmaceuticals team 👏 ! #RNAi #RNAtherapeutics #amyloidosis #cardiomyopathy
We are excited to share that the U.S. FDA has accepted our supplemental New Drug Application (sNDA) for vutrisiran, an investigational #RNAi therapeutic in development for the treatment of #ATTR-CM. https://bit.ly/412lQJn #RNAiTherapeutics #RNAinterference #amyloidosis
Senior Director, Engineering
2moI have a cousin who died of heart failure from this disease in his 40s very unexpectedly in 2005. He was the founder of Work Out World. Being the designer of a commercial oligonucleotide facility, I am so pleased to see an siRNA drug which might spare another person like Tony, who could not be helped. Seeing that disease treated is personal to the whole family.