Onpattro
Onpattro is a prescription medication used to treat polyneuropathy (nerve damage) in adults with hereditary transthyretin-mediated amyloidosis (hATTR), a rare genetic condition that causes abnormal protein buildup in organs and nerves. It is administered as an intravenous infusion and works by reducing the production of the abnormal transthyretin protein that causes the disease.
Onpattro is an RNAi therapeutic used to treat nerve damage in hereditary transthyretin-mediated amyloidosis by reducing abnormal protein production.
Key facts at a glance
- RxNorm
- 2053525
- Generic
- Onpattro
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Onpattro is indicated for the treatment of polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a rare inherited condition where abnormal transthyretin proteins build up in tissues throughout the body, causing nerve damage and organ dysfunction.
Onpattro is a small interfering RNA (siRNA) therapeutic that uses a lipid nanoparticle delivery system to target and silence the TTR gene in liver cells, reducing the production of abnormal transthyretin protein that causes amyloid deposits in nerves and organs.
Onpattro is administered by a healthcare provider as an intravenous infusion every 3 weeks. Common side effects may include infusion-related reactions, edema, and vitamin A deficiency. Patients should be monitored for liver abnormalities and allergic reactions during treatment. Regular follow-up with a healthcare provider experienced in amyloidosis is essential.
Official drug label
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This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy