Hereditary transthyretin amyloidosis progressively damages nerves through misfolded protein deposits, and silencing the source protein proved an effective strategy. Amvuttra (vutrisiran) refined that approach into infrequent dosing.
As an RNA interference therapy, it degrades transthyretin messenger RNA in the liver, reducing production of the protein that forms amyloid deposits. The subcutaneous injection is given once every three months for the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Amvuttra has since been approved for transthyretin amyloidosis with cardiomyopathy, a far larger population than the polyneuropathy indication. Quarterly self-administration differentiates it from its predecessor patisiran, and it competes with TTR stabilizers and emerging gene-editing approaches.
