Pronunciation: am-vutt-tra
Generic name: vutrisiran
Dosage form: single-dose prefilled syringe (25 mg/0.5 mL)
Drug class: Miscellaneous metabolic agents

Medically reviewed by Carmen Pope, BPharm. Last updated on May 9, 2025.

What is Amvuttra?

Amvuttra is used to treat polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR-PN) and cardiomyopathy associated with wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM). It is given as a subcutaneous (under the skin) injection every 3 months by a healthcare provider.

• When used to treat ATTR-CM, Amvuttra is used to reduce cardiovascular mortality, cardiovascular hospitalizations, and urgent heart failure visits.

Amvuttra (vutrisiran) gained FDA approval on June 13, 2022. There is no generic.

Mechanism

Amvuttra works by blocking the production of a protein called TTR (transthyretin). In transthyretin-mediated amyloidosis, mutated TTR proteins misfold, forming toxic amyloid deposits in nerves, the heart, and other organs.

Amvuttra (vutrisiran) contains a small piece of genetic material (siRNA) attached to a sugar molecule that helps it target liver cells where TTR is made. Once inside these cells, Amvuttra breaks down the genetic instructions for making TTR, reducing levels of TTR in the bloodstream and deposits of TTR in the tissues.

How effective is Amvuttra?

After 9 months of Amvuttra treatment (3 doses), clinical trials report that patients showed significant improvements in nerve function and quality of life. At 18 months:

• 48% of Amvuttra patients regained some nerve function vs. only 4% with placebo
• Remaining Amvuttra patients showed slowed neuropathy progression, while placebo patients worsened
• Amvuttra patients demonstrated improved walking speed, nutritional health, and daily activity performance.

Side effects

The most common side effects of Amvuttra are:

• pain in the hands and feet
• pain in the joints
• feeling short of breath
• low vitamin A levels
• pain, swelling, or redness where the injection was given.

Serious side effects and warnings

Amvuttra may cause the following serious side effects:

• Low vitamin A levels. Treatment with Amvuttra lowers the amount of vitamin A in your blood. Your doctor will tell you to take a vitamin A supplement every day. You should not take more than the amount of vitamin A recommended by your doctor. Low vitamin A levels can affect vision. If you have problems with your vision (e.g., night blindness) while taking Amvuttra, talk to your doctor. Your doctor may refer you to an eye specialist.
• Harm to an unborn baby. Amvuttra decreases vitamin A levels and may harm an unborn baby. Talk to your healthcare provider if you are pregnant, planning to become pregnant, or get pregnant while taking Amvuttra.

Amvuttra is for adults only. It is not known if it is safe and effective in children.

This is not a complete list of side effects. Call your doctor for medical advice about side effects. You may report side effects to Alnylam Pharmaceuticals at 1-877-256-9526 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Related/similar drugs

Before receiving

Before receiving Amvuttra, tell your doctor about all your medical conditions or allergies. Especially tell your doctor if you are: 

• Pregnant or intending to become pregnant
• Breastfeeding.

Pregnancy

Amvuttra may harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant. If you are pregnant, your name may be listed on a pregnancy registry to track the effects of Amvuttra on the baby.

Amvuttra can cause low vitamin A levels. Your doctor may have you take vitamin A while you are using Amvuttra. Take only the amount of vitamin A your doctor has prescribed. Vitamin A deficiency or excess in expectant mothers can cause vision problems, CNS deformities, and other serious side effects in the unborn baby.

Breastfeeding

It may not be safe to breastfeed while using this medicine. Ask your doctor about any risks.

How is Amvuttra administered?

Amvuttra is given by a healthcare provider via subcutaneous (under the skin, SC) injection. 

• It is administered once every 3 months (4 times a year).
• Amvuttra can be administered in the abdomen (but not within 2 inches of your belly button), upper thighs, or the back of the upper arms.
• Tell your caregivers if you feel any burning, pain, or swelling at the site where Amvuttra is injected.

Your doctor may have you take vitamin A while you are using Amvuttra.

• Take only the amount of vitamin A your doctor has prescribed.
• Call your doctor immediately if you have vision problems (especially at night) while receiving Amvuttra.

Dosing information

Dosage for hATTR-PN or ATTR-CM: Amvuttra 25 mg SC once every 3 months.

What happens if I miss a dose?

If you miss your appointment for your Amvuttra injection, ring your healthcare provider as soon as you can for another appointment so Amvuttra can be administered as soon as possible.

Receive the next dose 3 months after the most recently administered dose.

What other drugs will affect this medicine?

Other drugs may interact with Amvuttra, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

No formal clinical drug interaction studies have been performed, but Amvuttra is not expected to cause drug-drug interactions or to be affected by inhibitors or inducers of cytochrome P450 enzymes.

See the Amvuttra package insert for more information about drug interactions.

Storage

Store in the refrigerator at 2°C to 30°C (36°F to 86°F) in the original carton until ready to use. Do not freeze.

Ingredients

Active ingredients: vutrisiran 25 mg in 0.5 mL

Inactive ingredients: water; sodium hydroxide; phosphoric acid; sodium phosphate, monobasic, dihydrate; sodium phosphate, dibasic, dihydrate; sodium chloride.

Available as a prefilled syringe containing vutrisiran 25 mg/0.5 mL.

Manufacturer

Amvuttra is made by Alnylam Pharmaceuticals, based in Cambridge, Massachusetts, USA. Alnylam specializes in RNAi therapeutics.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer