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Overview: Onpattro is used to treat nerve damage from a rare genetic condition called hereditary transthyretin amyloidosis (hATTR). It is given by a health care provider as an infusion into a vein (IV). Common side effects include infusion reactions. Tell your health care provider right away if you have symptoms of an infusion reaction, such as flushing, shortness of breath, chest pain, rash, or if you feel lightheaded or dizzy. Onpattro may also cause low vitamin A levels. Be sure to tell your health care provider about all supplements and vitamins you take, and follow your health care provider’s instructions about whether to take a vitamin A supplement.

Uses

What is Onpattro used for?

Onpattro (patisiran) is commonly used to treat nerve damage (polyneuropathy) caused by hereditary transthyretin amyloidosis (hATTR). hATTR is a rare genetic condition.

Treating Polyneuropathy of Hereditary Transthyretin Amyloidosis (hATTR) With Onpattro

Onpattro may also be used for other conditions as determined by your health care provider.

How does Onpattro work (mechanism of action)?

People with hereditary transthyretin amyloidosis (hATTR) have a gene mutation that causes the liver to make abnormal forms of a protein called transthyretin. The abnormal transthyretin proteins clump together and form deposits, called amyloids. These amyloid deposits can build up and damage tissues, nerves, and organs, which can lead to serious problems and death.

Onpattro is a type of RNA interference (RNAi) medicine. It interferes with and causes the breakdown of a type of RNA that our body needs to make transthyretin proteins. By doing this, Onpattro helps lower the amount of both normal and abnormal transthyretin. This can help lower the buildup of amyloid deposits.

How is Onpattro supplied (dosage forms)?

Onpattro is available in an injectable form that is given by a health care provider.

How should I store Onpattro?

Onpattro is usually given by a health care provider in a hospital or clinic. If you will store Onpattro at home, follow the instructions given by your health care provider.

Side Effects

What are the most common side effects of Onpattro?

The most common side effects of Onpattro are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Nose or throat infection, sore throat, or common cold symptoms
  • Infusion reactions (see below)

There may be other side effects of Onpattro that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Onpattro?

While less common, the most serious side effects of Onpattro are described below, along with what to do if they happen.

Severe Allergic Reactions. Onpattro may cause allergic reactions, which can be serious. Get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Infusion Reactions. Onpattro can cause reactions that can be serious when it is given to you. Your infusion may need to be slowed or stopped if you have these reactions. Tell your health care provider right away if you have any of the following symptoms when you get your infusion. 

  • Redness, warmth, or swelling of your face and neck
  • Back pain
  • Nausea or vomiting
  • Stomach, joint, or muscle pain
  • Shortness of breath or trouble breathing
  • Headache
  • Feeling dizzy, lightheaded, or fainting
  • Fever or chills
  • Chest pain
  • Fast heartbeat
  • Hives or rash

Low Vitamin A Levels. Onpattro may lower your vitamin A levels and lead to a vitamin A deficiency. Follow your health care provider’s instructions about how much vitamin A to get through your diet and whether to take a vitamin A supplement. Tell your health care provider about all medicines, vitamins, and supplements you take, including multivitamins. Do not take more vitamin A than your health care provider recommends. Tell your health care provider right away if you have any symptoms of a vitamin A deficiency, such as trouble seeing in low light (night blindness) or any other changes to your eyes or vision, such as hazy vision, dry or crusty eyes, or clouding in your eyes.

Warnings & Precautions

Who should not use Onpattro?

Allergies to Ingredients. People who are allergic to any of the following should not use Onpattro.

  • Onpattro 
  • Patisiran
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Onpattro.

What should I know about Onpattro before using it?

Onpattro is usually given as an infusion into a vein (IV) by a health care provider in a hospital or clinic.

Your health care provider may give you medicines to help reduce the risk of certain side effects from Onpattro. Take these medicines exactly as your health care provider recommends.

What should I tell my health care provider before using Onpattro?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Onpattro is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your health care provider if you have any of the following.

  • Kidney problems
  • Liver problems
  • Low vitamin A levels

Pregnancy. It is not known if or how Onpattro could affect pregnancy or harm a fetus. Tell your health care provider right away if you are pregnant or plan to become pregnant. If you become pregnant while using Onpattro, you can sign up for a pregnancy registry by calling 877-256-9526 or by emailing [email protected]. This registry collects safety information about the use of Onpattro during pregnancy.

Breastfeeding. It is not known if Onpattro passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should use Onpattro while breastfeeding.

How can I get more information about Onpattro?

For more information about Onpattro, you can visit the drugmaker’s website at www.onpattro.com or call them at 833-256-2748.

Interactions

Does Onpattro interact with foods or drinks?

There are no known interactions between Onpattro and any foods or drinks.

It is unknown if drinking alcohol will affect Onpattro.

Does Onpattro interact with other medicines (drug interactions)?

There are no known interactions between Onpattro and other medicines. Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Onpattro?

If you or someone else has used too much Onpattro, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Onpattro?

If you miss an appointment to get your Onpattro dose, call to make another appointment as soon as possible. Your dosing schedule may need to be changed depending on how long it has been since you missed your dose.

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