Amvuttra (vutrisiran)
An approved treatment for ATTR Amyloidosis and Wild-Type ATTR Cardiac Amyloidosis.
The same compound appears under different names depending on the context. Here is how to identify Vutrisiran wherever you encounter it, plus the key facts at a glance.
- Generic name
- Vutrisiran
- Brand name
- Amvuttra
- Development code
- ALN-TTRSC02
- Drug class
- siRNA (small interfering RNA)
- Manufacturer
- Alnylam Pharmaceuticals
- How it's taken
- Vutrisiran is given as a 25 mg injection under the skin (subcutaneous) once every 3 months.
A next-generation RNA interference therapy for ATTR amyloidosis. Unlike patisiran (which requires IV infusion), vutrisiran is a single injection under the skin given once every 3 months.
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Where Vutrisiran fits
Second-generation RNAi therapy for ATTR with quarterly subcutaneous dosing. As of September 2026, the only TTR-silencing therapy approved for both polyneuropathy (hATTR-PN) and cardiomyopathy (ATTR-CM, wild-type and hereditary), positioning it as a direct alternative to TTR stabilizers like tafamidis and acoramidis for cardiac patients.
How Vutrisiran works
Vutrisiran sharply lowers how much TTR (transthyretin) protein your body makes. Like patisiran, it silences the genetic code for TTR. The key difference is that vutrisiran is chemically modified to be more stable in the body, so you need fewer doses: just 1 injection every 3 months instead of an IV infusion every 3 weeks.
Mechanism: RNA interference therapy that silences TTR gene expression
Side effects and safety
Vutrisiran lowers vitamin A levels in the blood, so the label advises taking a vitamin A supplement at the recommended daily amount (not higher). Tell your doctor about eye problems such as trouble seeing at night, which can be a sign of low vitamin A. The most common side effects in studies were pain in the arms or legs, joint pain, shortness of breath, and low vitamin A levels. Injection site reactions can also occur.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Vutrisiran
Vutrisiran is given as a 25 mg injection under the skin (subcutaneous) once every 3 months. This is a major advantage over patisiran, which requires IV infusions every 3 weeks. It is not a self-injection: a healthcare professional gives it at a doctor's office or treatment center, or in some cases at home if your insurance covers it.
Availability and cost
Only available as the brand-name product.
Next-generation RNAi therapy with an enhanced delivery platform allowing quarterly subcutaneous injections instead of triweekly IV infusions, reducing treatment burden but maintaining specialty pricing.
Help paying for Amvuttra
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
The Alnylam Assist Copay Program covers certain out-of-pocket costs for eligible commercially insured patients. Not for Medicare, Medicaid or other government insurance.
For: private insurance · source - Bridge or quick-start supply
Programs may help if you have a delay or change in insurance coverage; a Case Manager checks eligibility after a Start Form.
The official page does not say who qualifies. Ask the program. · source - Free medicine program
The Alnylam Assist Patient Assistance Program provides treatment at no cost for eligible patients, mainly those who are uninsured.
For: no insurance · source - Insurance and case manager help
After your doctor submits a Start Form, a Case Manager contacts you within 2 business days with a summary of your insurance benefits.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Good to know: Most services start when your doctor submits the Alnylam Assist Start Form. Alnylam can also point uninsured or underinsured patients to independent foundations.
- From a charity · HealthWell FoundationAmyloidosis fundOpen
Pays for: Copays, premiums or other treatment costs.
- From a charity · HealthWell FoundationCardiomyopathy (Medicare Access) fundOpen
Pays for: Copays, premiums or other treatment costs. Medicare patients only.
- From a charity · NORD RareCareAmyloidosis Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting Applications” - From a charity · NORD RareCareAmyloidosis Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting Applications” - From a charity · TotalAssist (formerly PAN Foundation)Amyloidosis fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $5,500 per year. Requires Medicare, Medicaid or TRICARE.
- From a charity · The Assistance FundAmyloidosis fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
Clinical trial results
FDA approved vutrisiran in June 2022 for hereditary ATTR with polyneuropathy (hATTR-PN) based on the HELIOS-A Phase 3 trial. On March 20, 2025, the FDA approved a label expansion for transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM, both wild-type and hereditary) based on the HELIOS-B Phase 3 trial, which showed a 28% reduction in the composite of all-cause mortality and recurrent cardiovascular events over 36 months. This made Amvuttra the first RNAi therapy approved for ATTR-CM.
Development history
Developed by Alnylam Pharmaceuticals as the follow-up to patisiran, with chemistry optimized for subcutaneous administration and quarterly dosing. Originally FDA-approved in June 2022 for hATTR with polyneuropathy. Indication expanded on March 20, 2025 to include ATTR amyloidosis with cardiomyopathy (ATTR-CM), making Amvuttra the only therapy approved in the U.S. for both the polyneuropathy and cardiomyopathy presentations of ATTR.
Explore ATTR Amyloidosis and Wild-Type ATTR Cardiac Amyloidosis trials
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Common questions about Vutrisiran
▸What is Vutrisiran (Amvuttra)?
A next-generation RNA interference therapy for ATTR amyloidosis. Unlike patisiran (which requires IV infusion), vutrisiran is a single injection under the skin given once every 3 months.
▸How does Vutrisiran work?
Vutrisiran sharply lowers how much TTR (transthyretin) protein your body makes. Like patisiran, it silences the genetic code for TTR. The key difference is that vutrisiran is chemically modified to be more stable in the body, so you need fewer doses: just 1 injection every 3 months instead of an IV infusion every 3 weeks.
▸What are the side effects of Vutrisiran?
Vutrisiran lowers vitamin A levels in the blood, so the label advises taking a vitamin A supplement at the recommended daily amount (not higher). Tell your doctor about eye problems such as trouble seeing at night, which can be a sign of low vitamin A. The most common side effects in studies were pain in the arms or legs, joint pain, shortness of breath, and low vitamin A levels. Injection site reactions can also occur.
▸How is Vutrisiran taken?
Vutrisiran is given as a 25 mg injection under the skin (subcutaneous) once every 3 months. This is a major advantage over patisiran, which requires IV infusions every 3 weeks. It is not a self-injection: a healthcare professional gives it at a doctor's office or treatment center, or in some cases at home if your insurance covers it.
▸Is Vutrisiran FDA approved?
Yes, Vutrisiran (Amvuttra) is FDA approved (2022) for the treatment of ATTR Amyloidosis and Wild-Type ATTR Cardiac Amyloidosis.
▸How does vutrisiran compare to patisiran?
Both are RNA interference therapies from Alnylam that silence TTR production, but vutrisiran has significant practical advantages. Vutrisiran is given as a subcutaneous injection once every 3 months, while patisiran requires an IV infusion every 3 weeks with premedication. Vutrisiran uses an enhanced stabilization chemistry (ESC) that makes the molecule more stable, allowing the longer dosing interval. Vutrisiran lowered blood TTR by about 83% in its nerve trial, similar to patisiran, but no study has formally compared the 2 drugs on nerve or heart outcomes.
▸Is vutrisiran approved for both heart and nerve involvement?
Yes. Vutrisiran was originally approved in June 2022 for hereditary ATTR with polyneuropathy, and in March 2025 it received an expanded approval for ATTR amyloidosis with cardiomyopathy, covering both hereditary and wild-type forms. This makes Amvuttra the only RNAi therapy approved for both the nerve and heart manifestations of ATTR amyloidosis.
▸What did the HELIOS-B trial show?
The HELIOS-B Phase 3 trial demonstrated that vutrisiran reduced the composite of all-cause mortality and recurrent cardiovascular events by 28% over 36 months in ATTR cardiomyopathy patients. On average, blood TTR levels fell by more than 80%, similar to the earlier nerve trial. These results led to the expanded FDA approval for ATTR cardiomyopathy in March 2025.
▸Can I switch from patisiran to vutrisiran?
Yes. Many patients have transitioned from patisiran (IV every 3 weeks) to vutrisiran (subcutaneous every 3 months) for the convenience of less frequent dosing and no need for IV infusion. Your physician can guide the transition timing. Both drugs achieve similar levels of TTR suppression, so the switch is primarily about treatment convenience.
▸Does vutrisiran require premedication like patisiran?
No. Because vutrisiran is given as a subcutaneous injection rather than an IV infusion, it does not require the corticosteroid and antihistamine premedication that patisiran does. This is another practical advantage of vutrisiran, as it simplifies the treatment process and reduces time spent at the clinic.
▸How does vutrisiran compare to tafamidis for heart involvement?
Vutrisiran and tafamidis take different approaches to treating ATTR cardiomyopathy. Tafamidis stabilizes the TTR protein to prevent misfolding, while vutrisiran lowers TTR production by about 80% through gene silencing. Both have shown cardiovascular benefits in Phase 3 trials. Tafamidis has a longer track record (approved 2019) and is taken as a daily pill. Vutrisiran is an injection given by a healthcare professional once every 3 months. Head-to-head trials have not been conducted.