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Medically reviewed by Last updated on Jul 22, 2020.


(pat i SIR an)

Index Terms

  • Patisiran Sodium

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Solution, Intravenous [preservative free]:

Onpattro: 10 mg/5 mL (5 mL)

Brand Names: U.S.

  • Onpattro

Pharmacologic Category

  • Anti-Transthyretin Small Interfering Ribonucleic Acid (siRNA) Agent


Patisiran is a double-stranded small interfering ribonucleic acid (siRNA) that causes degradation of mutant and wild-type transthyretin (TTR) mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues. Serum TTR is a carrier of retinol binding protein, which is involved in the transport of vitamin A in the blood.


Vdss: 0.26 ± 0.2 L/kg


By nucleases to nucleotides of various lengths


Urine: <1% as unchanged drug

Onset of Action

10 to 14 days (mean serum TTR reduced by ~80%)

Half-Life Elimination

3.2 ± 1.8 days

Protein Binding

Plasma protein: ≤2.1% (albumin and human alpha1-acid glycoprotein)

Use: Labeled Indications

Polyneuropathy: Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.


There are no contraindications listed in the US manufacturer's labeling.

Canadian labeling: Severe hypersensitivity (eg, anaphylaxis) to patisiran or any component of the formulation.

Dosing: Adult

Note: At least 60 minutes prior to administration of patisiran, premedicate with a corticosteroid, acetaminophen, an H1 blocker, and an H2 blocker to reduce the risk of infusion-related reactions (IRR).

Polyneuropathy: IV: Base dose on actual body weight.

<100 kg: 0.3 mg/kg once every 3 weeks

≥100 kg: 30 mg once every 3 weeks

Missed dose:

Within 3 days of missed dose: Administer dose, then continue dosing according to original schedule.

More than 3 days after missed dose: Administer dose, then continue dosing every 3 weeks thereafter.

Dosing: Geriatric

Refer to adult dosing.

Dosing: Adjustment for Toxicity

Infusion-related reactions (IRR): Consider slowing or discontinuing the infusion and resume at a slower infusion rate when symptoms have resolved; consider additional or higher doses of premedication in these patients during subsequent infusions to reduce IRR risk. If a serious of life-threatening IRR occurs, discontinue and do not readminister.


Remove from refrigeration and allow to come to room temperature. Do not shake. Based on calculated dosage, withdraw the entire contents of one or more vials into a syringe. Filter through a sterile 0.45 micron polyethersulfone syringe filter into a sterile container. Withdraw the required volume of filtered solution using a syringe. Add to a DEHP-free infusion bag containing NS for a total volume of 200 mL. Gently invert the bag; do not shake. Do not mix or dilute with other drugs.


IV: Following dilution and filtering, administer through a dedicated free-flowing venous access line with a DEHP-free infusion set containing a 1.2 micron polyethersulfone (PES) in-line infusion filter. Infuse over ~80 minutes, with an initial infusion rate of 1 mL/minute for 15 minutes, then increase to ~3 mL/minute for the remainder of the infusion (may extend duration of infusion >80 minutes to manage or prevent infusion-related reactions. After infusion is complete, flush IV administration set with NS. Monitor infusion site for infiltration and manage suspected extravasation using local standard practice for nonvesicants.


Store intact vials at 2°C to 8°C (36°F to 46°F). Do not freeze. Discard if frozen. If refrigeration is not available, store at room temperature ≤25°C (≤77°F) for ≤14 days. After dilution and filtration, discard any unused drug. Administer immediately after preparation, or store in infusion bag at room temperature (≤30°C [≤86°F]) for up to 16 hours, including infusion time.

Drug Interactions

There are no known significant interactions.

Adverse Reactions

The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.


Respiratory: Upper respiratory tract infection (29%)

Miscellaneous: Infusion related reaction (19%)

1% to 10%:

Cardiovascular: Atrioventricular block (3%), flushing (>2%)

Central nervous system: Vertigo (5%)

Dermatologic: Erythema (7%)

Gastrointestinal: Dyspepsia (8%), abdominal pain (>2%), nausea (>2%)

Neuromuscular & skeletal: Muscle spasm (8%), arthralgia (7%), back pain (>2%)

Ophthalmic: Keratoconjunctivitis sicca (5%), blurred vision (3%), visual disturbance (2%)

Respiratory: Dyspnea (8%), bronchitis (7%)

<1%, postmarketing, and/or case reports: Hypotension, injection site extravasation, syncope, vitamin A deficiency


Concerns related to adverse effects:

• Infusion-related reactions: Infusion-related reactions (IRR) have been reported, with a majority occurring within the first 2 infusions and the frequency decreasing with additional infusions. Common symptoms included abdominal, back or chest pain, dyspnea, facial edema, flushing, headache, nausea, rash and tachycardia. Premedicate with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers). Monitor closely; if an IRR occurs, consider slowing or interrupting the infusion. If interrupted, wait until symptoms have resolved before resuming the infusion at a slower rate. With future infusions, consider a slower infusion rate or higher doses of premedications. If a serious IRR occurs, stop the infusion and do not resume.

• Reduced vitamin A levels: A decrease in serum vitamin A has been reported with patisiran treatment. Supplement at the recommended daily allowance (RDA) of vitamin A during treatment. Do not administer doses higher than the RDA; serum vitamin A levels do not reflect the total vitamin A in the body. If ocular symptoms such as night blindness develop, refer the patient to an ophthalmologist.

Monitoring Parameters

Infusion-related reactions; ocular symptoms indicative of vitamin A deficiency (eg, night blindness)

Pregnancy Considerations

Patisiran decreases serum concentrations of vitamin A, which is required for normal fetal development. The effects to the fetus of maternal vitamin A supplementation during patisiran therapy, or the effects to the fetus of reducing maternal serum transthyretin, are not known.

Data collection to monitor pregnancy and infant outcomes following exposure to patisiran is ongoing. Health care providers are encouraged to enroll females exposed to patisiran during pregnancy in the Pregnancy Registry (877-256-9526 or Pregnant women may also register themselves.

Patient Education

What is this drug used for?

• It is used to treat nerve problems in people with amyloidosis.

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Common cold symptoms

• Nausea

• Muscle spasm

• Joint pain

• Redness

• Dizziness

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Infusion reaction

• Vision changes

• Poor night vision

• Shortness of breath

• Severe injection site redness, burning, pain, swelling, or irritation

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a limited summary of general information about the medicine's uses from the patient education leaflet and is not intended to be comprehensive. This limited summary does NOT include all information available about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not intended to provide medical advice, diagnosis or treatment and does not replace information you receive from the healthcare provider. For a more detailed summary of information about the risks and benefits of using this medicine, please speak with your healthcare provider and review the entire patient education leaflet.

Frequently asked questions

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Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.