Patisiran
Patisiran is an RNAi therapeutic agent used to treat hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It works by delivering double-stranded small interfering RNA (siRNA) that targets and degrades TTR mRNA, reducing the production of abnormal transthyretin protein that causes amyloid deposits in tissues.
Patisiran is an siRNA therapy that reduces TTR protein production to treat hereditary ATTR amyloidosis.
Key facts at a glance
- ATC
- N07XX12
- RxNorm
- 2053490
- Generic
- Patisiran
- Class
- atc-n07
- Brands
- Onpattro
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- 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.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
9 groupspregnancy
first trimesteravoid if possible
use only if potential benefits outweigh potential risks to the fetus; advise women of reproductive potential to use effective contraception during treatment
regular fetal monitoring and ultrasound assessments
Use with caution and monitor for adverse effects.
pregnancy
second third trimesteravoid if possible
continue only if benefits outweigh risks; close obstetric monitoring recommended
fetal growth monitoring and regular prenatal checkups
Use with caution and monitor for adverse effects.
lactation
breastfeedingdiscontinue breastfeeding or discontinue drug
it is unknown whether patisiran is excreted in human breast milk; decision should be made based on benefit-risk assessment
monitor infant for any adverse effects
geriatric
elderly over 65no dose adjustment required
no specific dose adjustment needed based on pharmacokinetic data; monitor renal and hepatic function
regular monitoring of renal and hepatic function, vital signs during infusion
pediatric_age
children adolescentsnot recommended
patisiran is not approved for use in pediatric patients; safety and efficacy have not been established
not applicable
Use of this medication in this population requires careful consideration of risks and benefits.
renal_impairment
mild moderateno dose adjustment required
patisiran is not renally cleared; mild to moderate renal impairment does not significantly affect exposure
monitor renal function regularly
Use with caution and monitor for adverse effects.
renal_impairment
severeuse with caution
limited data in patients with severe renal impairment; monitor closely
close monitoring of renal function and adverse reactions
Use with caution and monitor for adverse effects.
hepatic_impairment
mildno dose adjustment required
patisiran is metabolized in liver; mild hepatic impairment did not significantly affect exposure
monitor hepatic function
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate severeuse with caution
patients with moderate to severe hepatic impairment were excluded from clinical trials; use only if benefits outweigh risks
close monitoring of hepatic function and clinical response
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
FDA approval information for Patisiran
Drug class
Drug classification information
Primary indication
Therapeutic indication
Mechanism of action
Pharmacological mechanism
Treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with polyneuropathy. This is a rare genetic condition where abnormal TTR protein forms amyloid deposits in various tissues including nerves, heart, and digestive system.
Patisiran contains double-stranded siRNA that specifically targets and degrades both mutant and wild-type TTR messenger RNA (mRNA) through RNA interference. This leads to decreased production of TTR protein in the liver, reducing serum TTR levels and subsequent amyloid deposits in tissues.
Administered as an intravenous infusion every 3 weeks by a healthcare professional. Premedication with corticosteroids, antihistamines, and H2 antagonists is required to reduce infusion-related reactions. Monitor liver function and obtain baseline ophthalmological assessments. May cause vitamin A deficiency; supplementation may be needed. Safety in pregnancy is not established.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
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