Vutrisiran

Pronunciation: voo-triss-sir-ran
Generic name: vutrisiran
Brand name: Amvuttra
Dosage form: prefilled syringe for subcutaneous injection
Drug class: Miscellaneous metabolic agents

Medically reviewed by Carmen Pope, BPharm. Last updated on May 8, 2024.

What is vutrisiran?

Vutrisiran (Amvuttra) is an injectable siRNA – GalNAc conjugate that may be used to treat polyneuropathy (damage of multiple nerves throughout the body) in adults with hereditary transthyretin-mediated amyloidosis (hATTR). hATTR occurs when your liver produces faulty transthyretin (TTR) proteins and TTR deposits accumulate in organs and tissues, most commonly the peripheral nerves.

Vutrisiran combines a double-stranded small interfering RNA (siRNA) linked to a GalNAc conjugate that gives the siRNA stability, enhanced potency, and a long duration of action. The conjugate targets liver hepatocytes, where the siRNA is taken up and binds to variant and wild-type TTR mRNA, causing their degradation. This decreases levels of TTR protein and TTR amyloid protein deposits in tissues.

Vutrisiran was FDA-approved on June 13, 2022, under the brand Amvuttra. There is no generic Amvuttra available.  Amvuttra (vutrisiran) is given by a healthcare provider 4 times a year.

Vutrisiran side effects

Vutrisiran reduces serum vitamin A levels. Your healthcare provider will talk to you about taking the recommended daily allowance of vitamin A. Do not take higher than recommended doses of vitamin A to try to achieve normal serum vitamin A levels during treatment, as serum vitamin A levels do not reflect the total vitamin A in the body. Talk to your healthcare provider who should refer you to an ophthalmologist if you develop eye symptoms suggestive of vitamin A deficiency, such as night blindness.

The most common side effects of vutrisiran occurring in at least 5% of people administered it were pain in the extremities (hands and feet), joint pain, shortness of breath, and low levels of vitamin A.

Mild and short-lived injection site reactions (pain, swelling, redness at the injection site) were reported in 4% of patients administered vutrisiran, and 2 people in clinical trials experienced atrioventricular block.

This is not a complete list of side effects and others may occur. 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

Vyndamax, Amvuttra, Vyndaqel, Onpattro, Tegsedi, Wainua, tafamidis

Before receiving vutrisiran

Before receiving vutrisiran 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

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, especially if you are pregnant. 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 once every 3 months.

• The recommended dosage is 25mg. Vutrisiran (Amvuttra) is available as a prefilled syringe of 25 mg that is ready to administer.
• 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 around the IV needle when vutrisiran is injected.
• Your doctor may have you take vitamin A while you are using vutrisiran. Take only the amount of vitamin A your doctor has prescribed, especially if you are pregnant. An overdose of vitamin A can cause vision problems or other serious side effects.
• Call your doctor at once if you have vision problems (especially at night) while receiving vutrisiran.

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 vutrisiran can be administered as soon as possible. Resume dosing every 3 months from the most recently administered dose.

What happens if I overdose?

Since vutrisiran is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

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 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.

Amvuttra 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.

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

Manufacturer

Alnylam Pharmaceuticals, Inc.

Further information

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

Medical Disclaimer