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What Is Amvuttra?

Amvuttra is a prescription medicine containing vutrisiran, a transthyretin-directed small interfering RNA. It is approved for adults with polyneuropathy from hereditary transthyretin-mediated amyloidosis and for adults with cardiomyopathy from wild-type or hereditary transthyretin-mediated amyloidosis. Amvuttra comes as a 25 mg subcutaneous injection given once every three months by a healthcare professional.

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What Is Amvuttra?

Amvuttra is a prescription medicine containing vutrisiran, a transthyretin-directed small interfering RNA. It is approved for adults with polyneuropathy from hereditary transthyretin-mediated amyloidosis and for adults with cardiomyopathy from wild-type or hereditary transthyretin-mediated amyloidosis. Amvuttra comes as a 25 mg subcutaneous injection given once every three months by a healthcare professional.

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How Does Amvuttra Lower Transthyretin?

Vutrisiran uses RNA interference to break down mutant and wild-type transthyretin messenger RNA in liver cells. This lowers production of transthyretin protein and reduces transthyretin deposits in tissues. The mechanism targets the source of circulating transthyretin rather than removing deposits directly from affected nerves or the heart.

Who Is Treated With Amvuttra?

Amvuttra is approved for adults with hereditary transthyretin-mediated amyloidosis who have polyneuropathy. It is also approved for adults with cardiomyopathy from wild-type or hereditary transthyretin-mediated amyloidosis to reduce cardiovascular mortality, cardiovascular hospitalizations, and urgent heart failure visits. The current U.S. label does not establish pediatric use.

How Is Amvuttra Given?

The recommended dose is 25 mg injected under the skin once every three months. A healthcare professional administers each dose. If a scheduled dose is missed, it is given as soon as possible, and the next three-month interval is counted from that dose. Amvuttra comes in a single-dose prefilled syringe containing 25 mg in 0.5 mL.

Why Is Vitamin A Monitored During Amvuttra Treatment?

Amvuttra lowers serum vitamin A levels. Patients are advised to take the recommended daily allowance of vitamin A during treatment. Higher vitamin A doses are not used to normalize blood levels. Eye symptoms linked to vitamin A deficiency, such as night blindness, call for medical review and referral to an ophthalmologist.

Frequently Asked Questions About Amvuttra

How often is Amvuttra given?

Amvuttra is given as a 25 mg subcutaneous injection once every three months by a healthcare professional.

Does Amvuttra treat hereditary ATTR polyneuropathy?

Yes. It is approved for polyneuropathy from hereditary transthyretin-mediated amyloidosis in adults.

Does Amvuttra treat ATTR cardiomyopathy?

Yes. It is approved for cardiomyopathy from wild-type or hereditary transthyretin-mediated amyloidosis in adults.

Why is vitamin A supplementation used with Amvuttra?

Amvuttra lowers serum vitamin A levels, so patients take the recommended daily allowance during treatment.

References

AMVUTTRA (vutrisiran) Prescribing Information. https://www.amvuttrahcp.com/sites/default/files/AMVUTTRA-vutrisiran-Prescribing-Information.pdf. Date Accessed September 14, 2026.

DailyMed - AMVUTTRA (vutrisiran) Injection. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8db0facb-81b6-4006-9239-27dc6409c5d3. Date Accessed September 14, 2026.

Drug Trials Snapshots: AMVUTTRA. https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trial-snapshots-amvuttra. Date Accessed September 14, 2026.

Search Orphan Drug Designations and Approvals - Vutrisiran. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=637618. Date Accessed September 14, 2026.

Vutrisiran Injection. MedlinePlus. https://medlineplus.gov/druginfo/meds/a622061.html. Date Accessed September 14, 2026.