Vutrisiran

Pronunciation: voo-triss-sir-ran
Generic name: vutrisiran
Brand name: Amvuttra
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 Jun 30, 2025.

What is vutrisiran?

Vutrisiran (brand name Amvuttra) is used to treat nerve damage associated with hereditary transthyretin-mediated amyloidosis (hATTR) or cardiomyopathy associated with either wild-type ATTR or hATTR, to reduce cardiovascular mortality, cardiovascular hospitalizations, or urgent heart failure visits. It is given every 3 months by subcutaneous (under the skin) injection by a healthcare provider.

Vutrisiran works by targeting transthyretin (TTR) production, which is a protein found in liver cells. In people with ATTR, mutations in the TTR gene lead to misfolded TTR proteins that accumulate as amyloid deposits throughout the body, particularly in the nerves and the heart. Vutrisiran significantly reduces levels of TTR, reducing amyloid deposits and relieving nerve and heart damage associated with ATTR.  

Vutrisiran belongs to the drug class called small interfering RNA (siRNA) molecules.

Vutrisiran gained FDA approval on June 13, 2022, under the brand Amvuttra. There is no generic. Approval for vutrisiran was based on positive results from the HELIOS-A trial (NCT03759379, n= 122 hATTR patients), which reported 48% regained some nerve function compared to only 4% with placebo, after 18 months. Even in those whose nerves didn't improve, vutrisiran slowed neuropathy progression.

• Supplemental approval for cardiomyopathy was supported by HELIOS-B (NCT04153149), which showed vutrisiran reduced the combined risk of death and cardiovascular events by 28% while preserving functional capacity and quality of life.

Side effects

The most common side effects of vutrisiran are:

• discomfort or pain in the hands or feet
• joint pain
• breathing difficulties 
• decreased vitamin A levels
• injection site reactions (pain, redness, or swelling).

Serious side effects and warnings

Vitamin A Deficiency: Vutrisiran reduces vitamin A concentration in the bloodstream. Your physician will prescribe daily vitamin A supplementation. It's important to take only the recommended dosage—no more, no less. Reduced vitamin A can impact vision. Contact your doctor if you experience visual changes (such as difficulty seeing in dim light) while on vutrisiran. A referral to an eye specialist may be necessary.

Pregnancy Risks: Due to its effect on vitamin A levels, vutrisiran may cause fetal harm. Consult with your healthcare provider about receiving this medication if you are pregnant, considering pregnancy, or become pregnant during treatment.

Vutrisiran is not approved for use in children.

Not all possible side effects are listed here. Contact your healthcare provider about any concerning symptoms. Adverse events can be reported to Alnylam Pharmaceuticals (1-877-256-9526) or the FDA (1-800-FDA-1088 or www.fda.gov/medwatch).

Related/similar drugs

Before receiving

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

• have low levels of vitamin A
• have arthritis or joint pain
• have a breathing disorder such as asthma or COPD
• are pregnant or intending to become pregnant
• are breastfeeding or intending to breastfeed.

Pregnancy

Vutrisiran 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 vutrisiran on the baby.

Vutrisiran can cause low vitamin A levels. Your doctor may have you take vitamin A while you are using vutrisiran. Take only the amount of vitamin A your doctor has prescribed. Vitamin A deficiency or excess in expectant mothers can cause vision problems or 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 vutrisiran administered?

Vutrisiran is given by a healthcare provider by subcutaneous (under the skin) injection

• It is administered once every 3 months (4 times a year).
• It can be administered in the abdomen (but not within 2 inches of the 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 of injection.

Your doctor may have you take vitamin A while you are taking this medicine. Take only the amount of vitamin A your doctor has prescribed. An overdose of vitamin A can cause vision problems or other serious side effects.

• Call your doctor at once if you develop vision problems (especially at night) while receiving vutrisiran.

Dosing information

• Recommended adult dosage of vutrisiran (Amvuttra): 25 mg SC.

What happens if I miss a dose?

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

Resume dosing every 3 months from the most recently administered dose.

What other drugs will affect vutrisiran?

Other drugs interact with vutrisiran, 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 vutrisiran is not expected to cause drug-drug interactions or to be affected by inhibitors or inducers of cytochrome P450 enzymes.

See the Amvuttra (vutrisiran) Package Insert for more information about drug interactions.

Storage

Your healthcare provider will store vutrisiran in the refrigerator at 2°C to 30°C (36°F to 86°F) in the original carton until ready to use. It should not be frozen.

Ingredients

Vutrisiran injection is only available as Amvuttra.

• 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.

Amvuttra is a prefilled drug delivery system containing vutrisiran 25 mg/0.5 mL.

Who makes vutrisiran?

Vutrisiran is made by Alnylam Pharmaceuticals, under the brand name Amvuttra. Alnylam is based in Cambridge, Massachusetts, USA.

Further information

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

Medical Disclaimer