NDAINTRAVENOUSSOLUTIONPriority Review
Approved
Aug 2018
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
11
Data completeness
5/8 — Partial
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. First approved in 2018.
Drug data last refreshed 3h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ONPATTRO (patisiran) is an intravenous RNAi therapeutic that reduces transthyretin (TTR) protein by degrading mutant and wild-type TTR mRNA through RNA interference. It treats hereditary transthyretin amyloidosis (hATTR), a rare, progressive, life-threatening disease. Patisiran addresses an unmet need in a small patient population with limited prior treatment options.
$9.0MPart D
Peak8.9 years to LOEONPATTRO is at peak commercial maturity with modest Part D spending, indicating a stable but niche franchise supporting a focused brand team in a specialized rare disease setting.
Mechanism 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.
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (11)
Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
Started Aug 2022
10 enrolled
PolyneuropathiesWild Type ATTR AmyloidosisWild-Type Transthyretin-Related (ATTR)Amyloidosis+2 more
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
Started Aug 2020
10 enrolled
Hereditary Transthyretin-mediated (hATTR) AmyloidosisPolyneuropathy
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
Started Dec 2019
67 enrolled
Hereditary Transthyretin-mediated (ATTRv) AmyloidosisPolyneuropathy
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Started Sep 2019
360 enrolled
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
Started Mar 2019
24 enrolled
Amyloidosis, FamilialTransthyretin 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.