NDASUBCUTANEOUSSOLUTION
Approved
Jun 2022
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
4
Data completeness
7/8 — Comprehensive
AMVUTTRA (vutrisiran) by Alnylam Pharmaceuticals is double-stranded sirna-galnac conjugate that causes degradation of mutant and wild-type ttr mrna through rna interference, which results in a reduction of serum ttr protein and ttr protein deposits in tissues. Approved for transthyretin amyloidosis, hereditary transthyretin-mediated amyloidosis with polyneuropathy, transthyretin amyloidosis with cardiomyopathy. First approved in 2022.
Drug data last refreshed 20h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
AMVUTTRA (vutrisiran) is a double-stranded siRNA-GalNAc conjugate approved in June 2022 for treating hereditary transthyretin-mediated amyloidosis (hATTR) in adults. It addresses both polyneuropathy (hATTR-PN) and cardiomyopathy (ATTR-CM) indications by degrading mutant and wild-type TTR mRNA through RNA interference, leading to reduced serum TTR protein and tissue deposits. Administered subcutaneously, it represents a first-in-class RNAi therapeutic for this rare, progressive genetic disorder. The product is currently at peak lifecycle stage with no direct mechanistic competitors in the hATTR space.
$0.1BPart D
10.2 years to LOEMechanism of Action
double-stranded siRNA-GalNAc conjugate that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.