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Onpattro (PATISIRAN SODIUM)
Onpattro works by silencing the production of abnormal TTR protein through RNA interference.
Onpattro (Patisiran Sodium) is a small interfering RNA (siRNA) drug developed by Alnylam Pharmaceuticals, targeting transthyretin (TTR) mRNA to treat polyneuropathy in amyloidosis and transthyretin familial amyloid polyneuropathy. It was FDA-approved in 2018 and remains a patented product with no generic manufacturers. Onpattro works by silencing the production of abnormal TTR protein, which accumulates in nerves and causes damage. Key safety considerations include infusion-related reactions and liver enzyme elevations. As a patented product, Onpattro is commercially available through its manufacturer.
At a glance
| Generic name | PATISIRAN SODIUM |
|---|---|
| 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
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 |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |