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What Is Onpattro and How Does It Work?

Home  /  Other Health Conditions   /  What Is Onpattro and How Does It Work?
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Onpattro is a prescription drug used to treat a nerve condition. Its generic name is patisiran. It comes in a liquid solution and is injected into the vein for intravenous (IV) administration.


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Before taking Onpattro, it is crucial to inform your doctor and pharmacist:

  • About your medical conditions, disease history, and allergies.
  • About any herbal drugs, OTC medicines, or prescription medications you are taking.
  • If you are pregnant, breastfeeding, or planning to become pregnant.


What Is Onpattro?

Onpattro is a prescription-only medication containing the active ingredient, patisiran. It is a double-stranded small interfering ribonucleic acid (siRNA) used to treat polyneuropathy of familial transthyretin-mediated amyloidosis (hATTR), a nerve condition that affects adults. Onpattro is a lipid complex injection that is delivered via intravenous infusion.


What Is It Used To Treat?

Hereditary transthyretin-mediated amyloidosis with polyneuropathy is an inherited, progressive, and rare, life-threatening disease that damages multiple nerves throughout the body. It is the result of an inherited genetic mutation that misfolds a protein called transthyretin (TTR) into clusters known as amyloid deposits. Onpattro is used to treat this nerve disease. 


How Does Onpattro Work?

Onpattro belongs to the class of medications known as RNAi agents. RNAi agents or RNA interface is a collection of small RNA molecules used to treat genetic disorders.

Transthyretin protein is a protein that is produced in our liver (TTR). Patients with hereditary transthyretin-mediated amyloidosis have a mutation in the gene that codes for the TTR protein. The mutated gene causes the protein to fold in an unusual shape. 

The TTR protein becomes clumped together and forms amyloid plaques due to this improper folding. The deposits travel throughout the body and damage the nerves and organs.

Onpattro inhibits the accumulation of amyloid deposits throughout the body in the nerves and prevents the liver from making abnormal mutated transthyretin proteins. 


How Is Onpattro Administered?

  • Onpattro should be taken exactly as prescribed by your doctor.
  • Onpattro is administered in a medical setting by a healthcare professional.
  • Patients are given pre-medications at least 60 minutes before the administration of Onpattro in order to reduce the possibility of adverse infusion-related responses or side effects.
  • If intravenous pre-medications are not tolerated, the patient may be given oral equivalents, or the pre-medication doses may be decreased. 
  • Onpattro is injected as an IV infusion directly into the veins.
  • The drug is commonly given by IV infusion once every 3 weeks until disease progression.
  • Onpattro should be administered slowly as an IV infusion. The medication commonly takes 80 minutes to infuse completely into the vein.


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Side Effects

Onpattro patient with headacheThe possible side effects of Onpattro include:

  • Stomach pain
  • Nausea
  • Dizziness
  • Headache
  • Back pain
  • Chest discomfort
  • Flushing of skin

The serious side effects of Onpattro may include:

  • Irregular, slow, or fast heartbeat
  • Fainting
  • Difficulty breathing
  • Swelling of the tongue, lips, face, or throat
  • Redness, pain, or swelling at the injection site

Minor adverse effects of Onpattro fade as your body adjusts to the medication. However, if you have severe adverse effects or if mild side effects become unpleasant, persistent, or severe, contact your doctor or pharmacist immediately.


Onpattro Drug Interactions

Onpattro may interact with vitamins, herbal treatments, and over-the-counter medications. As a result, before beginning Onpattro, you must inform your doctor and pharmacist about any medications you are taking, including vitamins and herbal supplements.



Onpattro is supplied as a liquid solution in a single-dose vial. It is available in 10 mg/5ml (2 mg/ml) strength.



Onpattro dose is prescribed according to body weight.

  • Onpattro is given intravenously every 3 weeks at a dose of 0.3 mg/kg to individuals who weigh less than 100 kg.
  • Onpattro is given intravenously every 3 weeks in doses of 30 mg to individuals who weigh more than 100 kg.
  • No dose adjustments are required for hepatic or renal impairment. 



The following medications are frequently used as premedications 60 minutes prior to the administration of Onpattro:

  • 500 mg acetaminophen (oral)
  • IV corticosteroids, like dexamethasone 10 mg
  • Intravenous H1 blocker such as 50 mg diphenhydramine
  • Intravenous H2 blocker, such as 50 mg ranitidine  


Onpattro vs. Tegsedi

Onpattro and Tegsedi are both prescription drugs used to treat adult patients with stage 1 or stage 2 polyneuropathy with hereditary transthyretin amyloidosis (hATTR). They function by lowering TTR protein levels and enhancing nerve-related symptoms and functioning.

Onpattro and Tegsedi differ in that Tegsedi, also known as inotersen, can be self-administered by the patient once a week, while Onpattro must be provided by a medical practitioner in a clinical environment every 3 weeks.

Tegsedi therapy has a black box warning for low platelet count (thrombocytopenia), and it requires continual monitoring. On the other hand, Onpattro treatment does not call for ongoing observation. 

Note: Do not change your prescriptions without consulting your doctor.


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The average cost of Onpattro 2 mg/ml intravenous solution is around $10,313 for a supply of 5 ml.

The cost depends on several factors, including:

  • The location and type of pharmacy you visit
  • Your health condition and disease history
  • The number of doses prescribed by your doctor
  • Whether or not your insurance covers the cost of Onpattro drug treatment

Copay assistance may bring down the cost of this medication.



What kind of drug is Onpattro?

Onpattro is indicated in adults for treating hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with stage 1 or stage 2 polyneuropathy. It is a medication that belongs to the class of RNAi drugs, sometimes known as RNA interfaces, and It works by reducing the number of abnormal transthyretin proteins the liver makes. 

How long is Onpattro treatment?

Onpattro treatment is given every 3 weeks via IV infusion for about 80 minutes. The duration of the treatment depends on the progression of the disease and your body’s response to the drug. 

Is Onpattro FDA-approved?

Yes. It is a U.S. FDA-approved medication for treating hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) in adults.



Onpattro is a medicine administered intravenously to treat familial transthyretin-mediated amyloid polyneuropathy (hATTR-PN). The condition is distinguished by an abnormal accumulation of a protein known as amyloid in the body’s nerves, organs, and tissues.

Onpattro works by decreasing the amount of TTR proteins produced by the liver, hence reducing the number of amyloid plaques.

IV or oral pre-medications such as histamine blockers, IV corticosteroids, or acetaminophen are given 60 minutes before treatment to reduce Onpattro’s intravenous side effects.


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