If you have a certain kind of polyneuropathy (widespread nerve damage), your doctor may prescribe Onpattro.

It’s a prescription drug that’s used in adults with polyneuropathy caused by hereditary ATTR (hATTR) amyloidosis. This is a rare condition that runs in families. With amyloidosis, you have buildups of abnormal proteins in your body.

To learn more about hATTR amyloidosis and how Onpattro is used for it, see the “What is Onpattro used for?” section below.

Onpattro basics

Onpattro is a prescription medication that contains the active drug patisiran. It comes as a liquid solution that’s given by healthcare professionals.

You’ll receive Onpattro as an intravenous (IV) infusion. (With an IV infusion, a medication is slowly injected into your vein over a period of time.)

There’s no generic form available for Onpattro. Instead it’s only available as the brand-name drug.

Read on to learn more about Onpattro’s uses, side effects, dosage, and more.

Costs of prescription drugs can vary depending on many factors. These factors include what your insurance plan covers and which pharmacy you use. To find current prices for Onpattro in your area, visit WellRx.com.

If you have questions about how to pay for your prescription, talk with your doctor or pharmacist. You can also visit the Onpattro manufacturer’s website to see if they have support options.

Get answers to some commonly asked questions about Onpattro.

Is Onpattro used for cardiomyopathy?

Cardiomyopathy (damage to your heart’s muscle tissue) is often caused by hereditary ATTR (hATTR) amyloidosis. And while Onpattro is used in people with hATTR amyloidosis, it’s not approved to treat cardiomyopathy.

Hereditary ATTR amyloidosis is a rare condition that runs in families. And with amyloidosis, you have buildups of abnormal proteins in your body.

Instead, Onpattro is approved to treat polyneuropathy that’s caused by hATTR amyloidosis. Polyneuropathy is widespread nerve damage in your body.

Onpattro is sometimes used off-label for cardiomyopathy that’s caused by hATTR amyloidosis. (Off-label drug use is when a drug is prescribed for a condition other than its approved use.)

A study to test Onpattro’s effectiveness for cardiomyopathy caused by hATTR amyloidosis is currently ongoing.

If you’d like to know about treatment options for cardiomyopathy, talk with your doctor.

How does Onpattro work?

Onpattro is used to treat polyneuropathy (widespread nerve damage) that’s caused by hereditary ATTR (hATTR) amyloidosis.

Hereditary ATTR amyloidosis is a rare condition that runs in families. With this condition, abnormal protein deposits build up in different parts of your body. This leads to a range of symptoms that include nerve damage.

Onpattro’s mechanism of action is to block the effect of the abnormal gene that causes hATTR amyloidosis.

The drug attaches to your messenger RNA (mRNA), causing it to break down. mRNA is genetic material that your body uses as a blueprint to make proteins.

So, Onpattro works by interfering with your body’s process of making the abnormal proteins that build up in hATTR amyloidosis. With fewer abnormal proteins building up, Onpattro may help to reduce symptoms or worsening of hATTR amyloidosis.

Like most drugs, Onpattro may cause mild or serious side effects. The lists below describe some of the more common side effects that Onpattro may cause. These lists don’t include all possible side effects.

Keep in mind that side effects of a drug can depend on:

  • your age
  • other health conditions you have
  • other medications you may be taking

Your doctor or pharmacist can tell you more about the potential side effects of Onpattro. They can also suggest ways to help reduce side effects.

Mild side effects

Here’s a short list of some of the mild side effects that Onpattro can cause. To learn about other mild side effects, talk with your doctor or pharmacist, or read Onpattro’s patient brochure.

Mild side effects* of Onpattro that have been reported include:

Mild side effects of many drugs may go away within a few days or weeks. But if they become bothersome, talk with your doctor or pharmacist.

* For more information on these side effects, see the “Side effect focus” section below.

Serious side effects

Serious side effects from Onpattro can occur, but they aren’t common. If you have serious side effects from Onpattro, call your doctor right away. However, if you think you’re having a medical emergency, you should call 911 or your emergency number.

Serious side effects of Onpattro that have been reported include:

Note: Allergic reaction wasn’t reported during studies of Onpattro. But it’s still possible with this drug.

* For more information on this side effect, see the “Side effect focus” section below.

Side effect focus

Learn more about some of the side effects Onpattro may cause.

Upper respiratory infections

Some people taking Onpattro may get an upper respiratory infection (URI). This was a common side effect in people who received Onpattro in studies.

Some examples of URIs include sinus infection and the common cold. And possible symptoms of URI may include:

  • runny or stuffy nose
  • sinus pressure or pain
  • sore throat
  • sneezing

What might help

URIs are usually mild. And over-the-counter (OTC) cold medications may be useful in easing their symptoms.

Some examples of these medications include:

  • Antihistamines, which can help ease sneezing, runny nose, and sore throat. Some options are loratadine (Claritin) and cetirizine (Zyrtec).
  • Saline nose sprays or rinses, which can help ease a stuffy nose.
  • Pain relievers, which can help relieve a sore throat or sinus discomfort. Some options include acetaminophen (Tylenol) and ibuprofen (Advil, Motrin).
  • Decongestants, which can help ease stuffy nose and sinus discomfort. Pseudoephedrine (Sudafed) is a common nasal decongestant.

Not all OTC cold medications are safe for everyone. Be sure to check with your doctor or pharmacist before taking any medications.

Natural remedies are also an option to help ease common cold symptoms.

If you’re unsure about which cold medications or natural remedies are safe for you, check with your doctor or pharmacist. And if your upper respiratory infection symptoms don’t improve with these treatments, or the symptoms become bothersome, talk with your doctor.

Infusion reactions

Onpattro is given as an intravenous (IV) infusion. (With an IV infusion, a medication is slowly injected into your vein over a period of time.)

Some people have mild or serious side effects during their IV infusion of Onpattro or shortly after the infusion.

The most common mild infusion reactions include:

  • flushing (temporary warmth, redness, or deepening of skin color)
  • nausea
  • trouble breathing
  • back or joint pain
  • belly pain
  • headache

For most people, mild infusion-related reactions lessen or go away over time as your body gets used to the drug.

Less commonly, some people develop serious reactions with Onpattro infusions. A few examples of serious infusion reactions include:

  • extravasation (drug leakage from your vein into the tissue around your infusion site), which may cause:
    • pain
    • burning
    • swelling
  • fast heart beat
  • increased or decreased blood pressure
  • chest pain
  • facial swelling
  • rash

What might help

About an hour before each Onpattro infusion, your doctor will give you several medications to help reduce or prevent infusion-related reactions.

Onpattro is given by a healthcare professional, and they’ll monitor you for infusion reactions with each dose. Tell your doctor or nurse right away if you notice any unusual symptoms.

If you have an infusion reaction, your healthcare professional may stop, pause, or slow down your Onpattro infusion. They may also give medications to you to treat the reaction. But this all depends on the severity of your reaction.

If you have questions about possible infusion reactions with Onpattro, talk with your doctor.

Heart block

Although it’s not very common, Onpattro may cause a serious side effect called heart block. This condition is a problem with your heart’s electrical signals. And it affects your heart rhythm.

Heart block was reported in a small number of people who took the drug in a clinical study.

Symptoms of heart block may include:

  • feeling dizzy or lightheaded
  • passing out (fainting)

It’s important to note that heart problems are often a symptom of hereditary ATTR (hATTR) amyloidosis. And Onpattro is used to treat polyneuropathy (widespread nerve damage) in adults with hATTR amyloidosis. (For more information about hATTR amyloidosis, see the “What is Onpattro used for?” section below.)

What might help

If you pass out, or feel like you’re going to, while you’re receiving Onpattro, tell your doctor or nurse right away. They’ll monitor your heart carefully to check for heart block.

If you develop heart block, your doctor may temporarily or permanently stop your Onpattro treatment. And they may treat your heart block with medication or other therapies.

If you have questions about your risk of heart block with Onpattro, talk with your doctor.

Allergic reaction

Some people may have an allergic reaction to Onpattro. While allergic reaction wasn’t reported in studies of Onpattro, it’s still possible.

Symptoms of a mild allergic reaction can include:

  • skin rash
  • itchiness
  • flushing (temporary warmth, redness, or deepening of skin color)

A more severe allergic reaction is rare but possible. Symptoms of a severe allergic reaction can include swelling under your skin, typically in your eyelids, lips, hands, or feet. They can also include swelling of your tongue, mouth, or throat, which can cause trouble breathing.

Call your doctor right away if you have an allergic reaction to Onpattro. But if you think you’re having a medical emergency, call 911 or your local emergency number.

Your doctor will explain how Onpattro will be given to you. They’ll also explain how much you’ll be given and how often. Be sure to follow your doctor’s instructions. Below are commonly used dosages, but always follow the dosage your doctor prescribes.

Receiving Onpattro

Onpattro comes as a liquid solution that’s given by healthcare professionals.

You’ll receive Onpattro as an intravenous (IV) infusion. (With an IV infusion, a medication is slowly injected into your vein over a period of time.)

An Onpattro infusion usually takes about 80 minutes.

You’ll likely receive Onpattro infusions at your doctor’s office or a clinic. Or, if a healthcare professional comes to your home, you may be able to receive your Onpattro infusion at home. Talk with your doctor to discuss the best plan for you.

Dosage

You’ll receive Onpattro infusions once every 3 weeks.

Your doctor will determine the dose you’ll be given based on your body weight.

Receiving Onpattro with other drugs

About an hour before each Onpattro infusion, you’ll receive medications to help reduce infusion-related reactions. To read about infusion reactions with Onpattro, see the “What are Onpattro’s side effects?” section above.

The medications given for this purpose usually include:

Also, while you’re taking Onpattro, your doctor will prescribe a vitamin A supplement. This is because Onpattro can cause your body’s vitamin A level to become too low. And low vitamin A levels can lead to vision problems, such as night blindness (trouble seeing in low light).

Take your vitamin A supplement as prescribed. And don’t take extra vitamin A without first checking with your doctor. This will help your body maintain the right level of vitamin A.

Questions about receiving Onpattro

Here are some answers to common questions about receiving Onpattro.

  • What if I miss a dose of Onpattro? It’s important not to miss your Onpattro infusion appointments, which will be scheduled once every 3 weeks. If you miss your appointment, call your doctor’s office right away to reschedule. If you aren’t able to reschedule within 3 days of your missed dose, your next infusion appointments may also have to be rescheduled. Be sure to put your treatment schedule on your calendar and set a reminder alert on your phone, if possible.
  • Will I need to use Onpattro long term? Yes, Onpattro is meant to be a long-term treatment. In a study of adults receiving Onpattro, the drug was given once every 3 weeks for 18 months. Talk with your doctor to see how long you’ll need to receive Onpattro.
  • Should I take Onpattro with food? Onpattro is given as an infusion into your vein. So it doesn’t matter whether you have a full or empty stomach when you get doses of the drug. Talk with your doctor to see whether or not you should eat before your infusion appointments.
  • How long does Onpattro take to work? Onpattro starts working within a few weeks to reduce abnormal protein production in your body. It may take a few months for you to notice any improvement in your symptoms. Some people taking Onpattro don’t notice any changes. But the drug can slow down the worsening of your condition. You may not notice your symptoms getting better. But they may not get any worse after a few months of Onpattro treatment.
Questions for your doctor

You may have questions about Onpattro and your treatment plan. It’s important to discuss all your concerns with your doctor.

Here are a few tips that might help guide your discussion:

  • Before your appointment, write down questions like:
    • How will Onpattro affect my body, mood, or lifestyle?
  • Bring someone with you to your appointment if doing so will help you feel more comfortable.
  • If you don’t understand something related to your condition or treatment, ask your doctor to explain it to you.

Remember, your doctor and other healthcare professionals are available to help you. And they want you to get the best care possible. So, don’t be afraid to ask questions or offer feedback on your treatment.

Onpattro and Tegsedi are both used in adults to treat polyneuropathy that’s caused by hereditary ATTR (hATTR) amyloidosis.

Polyneuropathy is widespread nerve damage in your body. And hATTR amyloidosis is a rare condition that runs in families. With amyloidosis, you have buildups of abnormal proteins in your body.

One of the main differences between these two drugs is how they’re given. Onpattro comes as a solution that’s given to you by a healthcare professional. You’ll receive Onpattro every 3 weeks as an intravenous (IV) infusion. (With an IV infusion, a medication is slowly injected into your vein over a period of time.)

Tegsedi, on the other hand, comes as a solution that’s given once every week as an injection under your skin. After your doctor shows you how to do so, you can give Tegsedi injections to yourself at home.

To learn more about Onpattro and Tegsedi, read this in-depth comparison. And be sure to talk with your doctor about which drug is right for you.

If you have a certain kind of polyneuropathy (widespread nerve damage), your doctor may prescribe Onpattro.

Onpattro is used in adults to treat polyneuropathy that’s caused by hereditary amyloid transthyretin-mediated (hATTR) amyloidosis.

Hereditary ATTR amyloidosis is a rare condition. It’s caused by a mutation (abnormal change) in a certain gene. Parents with this gene mutation may pass it on to their children.

With amyloidosis, you have a buildup of abnormal protein deposits that form throughout your body. These protein deposits can lead to many different symptoms, such as:

  • trouble walking
  • fatigue (lack of energy)
  • heart problems

Onpattro isn’t used to treat all of the symptoms that hATTR amyloidosis causes. Instead, it’s specifically used for polyneuropathy that occurs with this condition.

Symptoms of polyneuropathy that Onpattro may help to ease include:

  • nerve-related symptoms of the hands, feet, arms, and legs, such as:
  • nerve-related symptoms of the digestive system, such as:
    • severe constipation, diarrhea, nausea, vomiting, and weight loss
  • other nerve-related symptoms such as:

There are some important things to discuss with your doctor when considering Onpattro treatment, such as:

  • your overall health
  • any medical conditions you have

Interactions

Taking certain medications, vaccines, foods, and other things with a drug can sometimes affect how a drug works. These effects are called interactions.

There aren’t any known interactions between Onpattro and other medications, supplements, vaccines, or foods.

But to be safe, before taking Onpattro, be sure to tell your doctor about all medications you take, including prescription and over-the-counter types. Tell them about any vitamins, herbs, or supplements that you use. Sharing this information and providing updates to your doctors and pharmacists can help you avoid potential interactions.

If you have any questions about possible interactions with Onpattro, talk with your doctor or pharmacist.

Warnings

Onpattro may not be right for you if you have certain medical conditions or other factors that affect your health. Talk with your doctor about your health history before you take Onpattro. Factors to consider include:

  • Allergic reaction. If you’ve had an allergic reaction to Onpattro or any of its ingredients, you shouldn’t take Onpattro. Ask your doctor about other medications that might be better options for you.

Use with alcohol

There aren’t any known interactions between Onpattro and alcohol.

But drinking alcohol could worsen some symptoms of hereditary ATTR (hATTR) amyloidosis, which Onpattro is used to treat. (To learn about this condition, see the “What is Onpattro used for?” section above.)

Symptoms that may be worsened can include:

  • digestion problems, such as diarrhea
  • tiredness
  • sexual problems, such as low libido

Talk with your doctor if you have questions about drinking alcohol while you’re receiving Onpattro.

Pregnancy and breastfeeding

It isn’t known if Onpattro is safe to take during pregnancy or while breastfeeding.

Keep in mind that Onpattro can lower your vitamin A level. So your doctor will give you a vitamin A supplement while you’re taking Onpattro. The right amount of vitamin A is needed to help a fetus develop normally.

If you’re pregnant or breastfeeding, talk with your doctor before starting Onpattro. They’ll recommend the best treatment plan for you at this time.

If you are pregnant during Onpattro treatment, check with your doctor about enrolling in Onpattro’s pregnancy registry. (Pregnancy registries collect information about a drug’s use during pregnancy.) To learn more about this drug registry, call 877-256-9526.

Note: Onpattro is used in adults with hereditary ATTR (hATTR) amyloidosis. This is a rare condition that’s caused by a mutation (abnormal change) in a certain gene. Parents with this gene mutation may pass it on to their biological children. If you’re planning to become pregnant, it’s important to talk with your doctor. Discuss your plans to have a family with your doctor. They’ll explain the health risks and the chances of passing this condition on to your child.

If you have polyneuropathy (widespread nerve damage) from hereditary ATTR (hATTR) amyloidosis, your doctor may recommend Onpattro. (To learn about this condition, see the “What is Onpattro used for?” section above.)

Keep in mind that this drug isn’t used for all symptoms of hATTR amyloidosis. It’s only used for polyneuropathy from hATTR amyloidosis.

You can learn more about treatment options for amyloidosis in this article. There are also natural remedies that may help ease amyloidosis symptoms.

If you have questions about Onpattro, talk with your doctor or pharmacist. If you’re not sure what to ask, here are some questions to help get your conversation started:

  • Where and when will I receive my Onpattro treatments?
  • Is there anything I should do to prepare for my first Onpattro infusion?
  • Do my health conditions put me at higher risk for side effects with Onpattro?
  • If I start using Onpattro, should I continue taking my other medications for my condition?

Q:

Will I need to receive Onpattro treatments for the rest of my life?

Anonymous patient

A:

Most likely, yes.

There’s no cure for hereditary ATTR (hATTR) amyloidosis right now. But Onpattro can help manage this life-long condition and improve your quality of life.

If you and your doctor decide that Onpattro is safe and effective for you, you’ll likely take it long term to:

  • relieve certain symptoms of hATTR amyloidosis
  • slow down the worsening of hATTR amyloidosis

If you have questions about how long you should take Onpattro, talk with your doctor.

Dana Kim, PharmDAnswers represent the opinions of our medical experts. All content is strictly informational and should not be considered medical advice.
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Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or other healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.