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Onpattro (PATISIRAN)
Onpattro works by silencing the production of abnormal TTR protein using small interfering RNA.
At a glance
| Generic name | PATISIRAN |
|---|---|
| Sponsor | Alnylam Pharmaceuticals |
| Drug class | Small Interfering RNA |
| Target | transthyretin (TTR) mRNA |
| Modality | Oligonucleotide |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2018 |
| Annual revenue | 700 |
Mechanism of action
Patisiran is double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in reduction of serum TTR protein and TTR protein deposits in tissues.
Approved indications
- Polyneuropathy in amyloidosis
- Transthyretin familial amyloid polyneuropathy
Common side effects
- Upper respiratory tract infections
- Infusion-related reaction
- Dyspepsia
- Dyspnea
- Muscle spasms
- Arthralgia
- Erythema
- Bronchitis
- Vertigo
- Dry eye
- Blurred vision
- Vitreous floaters
Key clinical trials
- Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
- Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
- Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy (EARLY_PHASE1)
- APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) (PHASE3)
- HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) (PHASE3)
- Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran
- Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR (PHASE2)
- Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Onpattro CI brief — competitive landscape report
- Onpattro updates RSS · CI watch RSS
- Alnylam Pharmaceuticals portfolio CI