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Generic name: patisiran
Brand name: Onpattro
Dosage form: intravenous solution (2 mg/mL)
Drug class: Miscellaneous metabolic agents

Medically reviewed by  A Ras MD.

What is patisiran used for?

Patisiran is a prescription medicine that is used to treat nerve problems in people with amyloidosis.


ONPATTRO contains patisiran, a double-stranded small interfering ribonucleic acid (siRNA), formulated as a lipid complex for delivery to hepatocytes. Patisiran specifically binds to a genetically conserved sequence in the 3′ untranslated region (3’UTR) of mutant and wild-type transthyretin (TTR) messenger RNA (mRNA).

The structural formula is:


A, adenosine; C, cytidine; G, guanosine; U, uridine; Cm, 2′-O-methylcytidine; Um, 2′-O-methyluridine; dT, thymidine

ONPATTRO is supplied as a sterile, preservative-free, white to off-white, opalescent, homogeneous solution for intravenous infusion in a single-dose glass vial. Each 1 mL of solution contains 2 mg of patisiran (equivalent to 2.1 mg of patisiran sodium). Each 1 mL also contains 6.2 mg cholesterol USP, 13.0 mg (6Z,9Z,28Z,31Z)-heptatriaconta-6,9,28,31-tetraen-19-yl-4-(dimethylamino) butanoate (DLin-MC3-DMA), 3.3 mg 1,2-distearoyl-sn-glycero-3-phosphocholine (DSPC), 1.6 mg α-(3′-{[1,2-di(myristyloxy)propanoxy] carbonylamino}propyl)-ω-methoxy, polyoxyethylene (PEG2000-C-DMG), 0.2 mg potassium phosphate monobasic anhydrous NF, 8.8 mg sodium chloride USP, 2.3 mg sodium phosphate dibasic heptahydrate USP, and Water for Injection USP. The pH is ~7.0.

The molecular formula of patisiran sodium is C412 H480 N148 Na40 O290 P40 and the molecular weight is 14304 Da.

Mechanism of Action

Patisiran is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.

Before taking patisiran, tell your doctor:

  • If you are allergic to patisiran; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.

This medicine may interact with other drugs or health problems.

Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take patisiran with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

What are some things I need to know or do while I take patisiran?

  • Tell all of your health care providers that you take patisiran. This includes your doctors, nurses, pharmacists, and dentists.
  • Infusion reactions have happened with patisiran. Tell your doctor if you have back or neck pain, muscle pain, chest pain, fast heartbeat, heartbeat that does not feel normal, flushing, headache, shortness of breath, stomach pain or upset, swelling of the face, very bad dizziness, passing out, or any other effects during the infusion.
  • You may need to take vitamin A while taking patisiran. Be sure to follow what your doctor has told you.
  • Tell your doctor if you are pregnant or plan on getting pregnant. You will need to talk about the benefits and risks of using patisiran while you are pregnant.
  • Do not take more vitamin A than what your doctor told you to take. Taking too much vitamin A during pregnancy may cause birth defects. If you have questions, talk with your doctor.
  • Tell your doctor if you are breast-feeding. You will need to talk about any risks to your baby.

How is patisiran best taken?

Use patisiran as ordered by your doctor. Read all information given to you. Follow all instructions closely.

  • It is given as an infusion into a vein over a period of time.
  • Other drugs will be given with patisiran to help avoid side effects.

What do I do if I miss a dose?

  • Call your doctor to find out what to do.

What are the side effects of patisiran that I need to call my doctor about immediately?

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:

  • 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.
  • Change in eyesight.
  • Lowered night eyesight.
  • Shortness of breath.
  • This medicine may irritate the vein. If the drug leaks from the vein, it may also cause irritation around that area. Tell your nurse if you have any redness, burning, pain, swelling, or leaking of fluid where the drug is going into your body.

What are some other side effects of patisiran?

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:

  • Signs of a common cold.
  • Upset stomach.
  • Muscle spasm.
  • Joint pain.
  • Redness.
  • Dizziness.

These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-332-1088. You may also report side effects at https://www.fda.gov/medwatch.

If overdose is suspected:

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

How do I store and/or throw out patisiran?

  • If you need to store patisiran at home, talk with your doctor, nurse, or pharmacist about how to store it.



  • NDC 71336-1000-1
  • onpattro®
    lipid complex injection
  • 10 mg/5 mL
    (2 mg/mL)
  • Sterile Solution for
    Intravenous Infusion Only
  • Dilute Before Use
  • Single-Dose Vial
  • Discard Unused Portion
  • Rx Only


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