NDASUBCUTANEOUSSOLUTION
Approved
Jun 2022
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
4
Data completeness
6/8 — Strong
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. First approved in 2022.
Drug data last refreshed 20h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
AMVUTTRA (vutrisiran) is a subcutaneous siRNA-GalNAc conjugate that degrades mutant and wild-type TTR mRNA through RNA interference, reducing serum TTR protein and tissue deposits. It is indicated for transthyretin (TTR) amyloidosis, a rare progressive genetic disorder affecting the heart, nervous system, and kidneys. The drug represents a novel RNA interference approach to a previously difficult-to-treat rare disease.
$0.067BPart D
PeakGrowing
9.7 years to LOEStrong peak-stage adoption with 562 Part D claims in 2023 signals active patient population expansion and sustained commercial investment in the rare disease franchise.
Mechanism 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.
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (4)
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
Started Feb 2026
125 enrolled
Hereditary Transthyretin-Mediated Amyloidosis With PolyneuropathyhATTR-PN
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Started Dec 2024
700 enrolled
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Started Nov 2019
655 enrolled
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Started Feb 2019
164 enrolled
Amyloidosis, HereditaryTransthyretin Amyloidosis
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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.