Amvuttra is a brand-name injection that’s prescribed for nerve damage caused by a rare genetic condition called hereditary amyloidosis. Amvuttra contains the active ingredient vutrisiran.

Specifically, the Food and Drug Administration (FDA) has approved Amvuttra to treat polyneuropathy (damage to multiple nerves) caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults. For details about this condition, see “Amvuttra uses.”

Drug details

You’ll find key information about Amvuttra below.

  • Drug class: RNA interference therapy (sometimes called a gene silencer)
  • Drug form: solution in a prefilled syringe given by subcutaneous injection (an injection just under your skin)
  • Generic available? no
  • Prescription required? yes
  • Controlled substance? no
  • Year of FDA approval: 2022

Amvuttra is available only as a brand-name medication. It’s not currently available in generic form.

A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics usually cost less than brand-name drugs.

The following information describes Amvuttra dosages that are commonly used or recommended. However, your doctor will determine the best Amvuttra dosing schedule to fit your needs.

Drug form and strength

Amvuttra comes as a solution inside a single-dose prefilled syringe. A healthcare professional will give your doses at your doctor’s office or clinic. You’ll receive Amvuttra by subcutaneous injection in your upper arm, thigh, or abdomen.

Amvuttra is available in only one strength: 25 milligrams (mg) in 0.5 milliliters (mL) of solution.

Dosage for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis

Amvuttra may be prescribed to treat nerve damage caused by a type of amyloidosis. (For details, see “Amvuttra uses.”)

The recommended Amvuttra dosage for this condition in adults is 25 mg (1 injection) once every 3 months.

About Amvuttra treatment

Below you’ll find information about key dosage issues.

  • If you miss a dose: If you miss an appointment to receive a dose of Amvuttra, contact your doctor’s office right away to reschedule. You should receive the missed dose as soon as possible. Your next dose should be given 3 months after you receive the rescheduled dose.
  • Length of use: Amvuttra is meant to be a long-term treatment. If you and your doctor determine that Amvuttra is safe and effective for you, you’ll likely receive it long term.
  • Length of time to work: Amvuttra starts working in your body right after you receive your first injection. But you might not notice it working. The drug helps prevent nerve damage from getting worse. Some people may have improved nerve function and reduced symptoms after a few months of treatment, but others may not have reduced symptoms.

Amvuttra can cause mild or serious side effects. The following lists contain some of the key side effects that may occur during Amvuttra treatment. These lists do not include all possible side effects.

For more information about the possible side effects of Amvuttra, talk with your doctor or pharmacist. They can give you tips on how to manage any side effects that may be concerning or bothersome.

Note: The Food and Drug Administration (FDA) tracks side effects of drugs it has approved. If you would like to notify the FDA about a side effect you’ve had with Amvuttra, you can do so through MedWatch.

Mild side effects

Below is a partial list of mild side effects of Amvuttra. To learn about other mild side effects, talk with your doctor or pharmacist or view the drug’s prescribing information.

Mild side effects of Amvuttra can include:

Most of these side effects may go away within a few days to a couple of weeks. However, if they become more severe or don’t go away, talk with your doctor or pharmacist.

Serious side effects

Serious side effects from Amvuttra aren’t common, but they can occur. Call your doctor right away if you have serious side effects. Call 911 or your local emergency number if your symptoms feel life threatening or you think you’re having a medical emergency.

Serious side effects and their symptoms can include:

  • low vitamin A levels, which may include symptoms such as:
  • heart block (problems with the heart’s electrical signals), which may include symptoms such as:
    • palpitations
    • dizziness or lightheadedness
    • fainting
Allergic Reaction

For some people, Amvuttra injection can cause an allergic reaction. But this side effect wasn’t reported in the drug’s clinical trials.

In general, symptoms of allergic reaction can be mild or serious.

Ways to manage

For mild allergic reaction symptoms, such as a mild rash, call your doctor right away. They may recommend treatments to help manage your symptoms. They’ll also let you know whether you should keep receiving Amvuttra injections.

For severe allergic reaction symptoms, such as swelling or trouble breathing, call 911 or your local emergency number right away. These symptoms require immediate medical care because they can become life threatening. If you’ve had a serious allergic reaction to Amvuttra, your doctor may recommend a different medication instead.

The Food and Drug Administration (FDA) approves prescription drugs such as Amvuttra to treat certain conditions. Amvuttra may also be used off-label for other conditions. Off-label drug use is when an FDA-approved drug is prescribed for a purpose other than what it’s approved for.

Amvuttra for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis

Amvuttra is FDA approved to treat polyneuropathy (damage to multiple nerves) caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults. This condition is also called transthyretin amyloidosis.

hATTR amyloidosis is a rare, inherited condition that causes your body to make abnormal transthyretin (TTR) proteins.

TTR proteins usually transport vitamin A and thyroid hormones around your body in your bloodstream. However, if you have hATTR amyloidosis, the abnormal TTR proteins form amyloid deposits, which damage your nerves and your organs, such as your heart and kidneys.

The nerve damage can affect nerves throughout your body, including those that are responsible for sensation and movement control and those that are responsible for involuntary processes such as digestion and blood pressure regulation.

Symptoms of nerve damage caused by hATTR amyloidosis can include:

Amvuttra reduces the amount of abnormal proteins your body makes, so there’s less buildup of amyloid deposits in your nerves. (For details about this, see the “How Amvuttra works” section later in this article.)

Having less amyloid buildup helps prevent your nerve damage from getting worse. It can also improve your nerve function and help reduce symptoms of nerve damage. This can improve your quality of life.

Amvuttra and children

Amvuttra is not FDA approved for use in children. It’s not known whether the drug is safe or effective in children.

Amvuttra is not known to interact with other medications, herbs, supplements, foods, or alcohol. The manufacturer did not list any interactions in the drug’s prescribing information.

However, this doesn’t mean that interactions won’t be recognized in the future. For example, new drugs could be approved that interact with Amvuttra.

Before starting Amvuttra treatment, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also, tell them about any vitamins, herbs, and supplements you take. Sharing this information can help you avoid potential interactions.

If you have questions about drug interactions that may affect you, talk with your doctor or pharmacist.

As with all medications, the cost of Amvuttra can vary. The actual price you’ll pay depends on your insurance plan, your location, and the pharmacy you use.

Financial and insurance assistance: If you need financial support to pay for Amvuttra or need help understanding your insurance coverage, help is available.

A program called Alnylam Assist is available for Amvuttra. For more information and to find out whether you’re eligible for support, call 833-256-2748 or visit the program website. If you’re prescribed Amvuttra, an Alnylam case manager will reach out to you after they receive a start form from your doctor.

Generic version: Amvuttra is not available in a generic form. A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics tend to cost less than brand-name drugs.

Learn more about saving money on prescriptions.

Other drugs are available that can treat your condition. Some may be a better fit for you than others. If you’re interested in finding an alternative to Amvuttra, talk with your doctor. They can tell you about other medications that may work well for you.

The following drugs are similar to Amvuttra:

For information about how Amvuttra and Onpattro compare, see the “Amvuttra vs. Onpattro” section.

If you can become pregnant, consider the following information about pregnancy, birth control, and breastfeeding.

Amvuttra and pregnancy

It’s not known whether Amvuttra should be used during pregnancy. The drug has not been studied in people who are pregnant.

Note that Amvuttra can lower vitamin A levels, so your doctor will usually recommend taking a vitamin A supplement during your Amvuttra treatment. However, it’s not known whether this is safe to do while pregnant.

Your body needs vitamin A to help a fetus develop. But if your vitamin A level gets too high, it can cause fetal harm. It’s not known whether changes to your vitamin A levels caused by Amvuttra and vitamin A supplements will affect a developing fetus.

If you’re planning a pregnancy or can become pregnant, talk with your doctor before starting Amvuttra.

Amvuttra and birth control

It’s not known whether Amvuttra is safe to use during pregnancy. If you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control needs during Amvuttra treatment.

Amvuttra and breastfeeding

It’s not known whether Amvuttra should be used while breastfeeding. If you’re currently breastfeeding or planning to do so, talk with your doctor before starting this medication.

You may wonder how Amvuttra compares with other medications that are prescribed for similar uses. Here we look at how Amvuttra compares to Onpattro.

Amvuttra and Onpattro are both used to treat polyneuropathy (damage to multiple nerves) caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). They belong to the same group of drugs (RNA interference therapies, or gene silencers) but contain different active ingredients. Amvuttra contains vutrisiran, and Onpattro contains patisiran.

Amvuttra is given by subcutaneous injection once every 3 months. Onpattro is given by intravenous (IV) infusion once every 3 weeks.

These drugs can cause some similar side effects, such as low vitamin A levels, joint pain, and shortness of breath. But they can also cause some different side effects. For example, Onpattro can cause upper respiratory infections and infusion reactions.

To find out more about how these medications compare, talk with your doctor. They can help determine whether either treatment is a good option for your condition.

Amvuttra is used to treat polyneuropathy (damage to multiple nerves) caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).

What happens with hATTR amyloidosis

If you have hATTR amyloidosis (also called transthyretin amyloidosis), you have an inherited genetic mutation (an abnormal change in a gene). This mutation causes your liver to make abnormal transthyretin (TTR) proteins, which form amyloid deposits in your nerves and organs. These deposits damage your nerves and organs.

The nerve damage can affect nerves that are responsible for sensation and movement control. This can cause symptoms such as numbness, tingling, burning pain, and muscle weakness.

The damage can also affect nerves that are responsible for involuntary processes such as blood pressure control and digestion. This can cause symptoms such as dizziness upon standing and constipation. (To learn more about hATTR symptoms, see the “Amvuttra uses” section.)

Amvuttra’s mechanism of action

Amvuttra works by blocking the effect of a gene mutation that causes hATTR amyloidosis. Specifically, Amvuttra is a type of drug called an RNA interference therapy (sometimes called a gene silencer).

Your genes carry instructions for making particular proteins. Inside your cells, a molecule called messenger RNA (mRNA) copies instructions from your genes and takes them to a part of the cell that builds proteins.

Amvuttra works by attaching to mRNA that has copied instructions for making abnormal TTR proteins. This causes the mRNA to break down, which stops cells in your liver from making as many abnormal TTR proteins.

By reducing the production of abnormal TTR proteins, Amvuttra reduces the buildup of amyloid deposits in your nerves. This helps prevent nerve damage from getting worse. Your body may also break down existing deposits over time, which can improve your nerve function and help reduce symptoms of nerve damage.

If you’d like to know more about how Amvuttra works, talk with your doctor. You can also refer to the drug’s prescribing information or visit the manufacturer’s website.

Before starting Amvuttra, discuss your health history with your doctor. Amvuttra may not be right for you if you have certain medical conditions or other factors affecting your health. Be sure to talk with your doctor if any of the following apply to you:

Note: For more information about the potential negative effects of Amvuttra, see the “Amvuttra side effects” section.

Disclaimer: Medical News Today 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 another 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.