NDAINTRAVENOUSSOLUTIONPriority Review
Approved
Aug 2018
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
12
Data completeness
6/8 — Strong
Priority Review
ONPATTRO (patisiran) by Alnylam Pharmaceuticals is double-stranded sirna 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, transthyretin amyloidosis with cardiomyopathy, familial amyloid polyneuropathy. First approved in 2018.
Drug data last refreshed 20h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ONPATTRO (patisiran) is an intravenous siRNA therapeutic developed by Alnylam Pharmaceuticals for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It works by degrading both mutant and wild-type transthyretin (TTR) mRNA through RNA interference, resulting in reduced serum TTR protein and decreased tissue deposition. As the first FDA-approved RNAi therapeutic for this indication, it represents a paradigm shift in treating a previously difficult-to-manage rare genetic disorder.
$0.0BPart D
9.3 years to LOEMechanism of Action
double-stranded siRNA 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.