Vutrisiran Injection
Vutrisiran is an RNA-based injection used to treat nerve damage and heart muscle disease caused by transthyretin-mediated amyloidosis (a protein-buildup disorder).
🧬 Where this information comes from Click to expand
The clinical label records used on this page are FDA-filed Structured Product Labeling (SPL) retrieved through openFDA. We identified 1 clinical label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. Forms, routes, brands, labelers, strengths, and NDC product-record counts are built separately from matching FDA National Drug Code Directory records. The linked DailyMed document is a representative official source for verification, not the page’s only clinical source. View the linked label on DailyMed →
📖 Vutrisiran Injection: Patient Information Guide
A plain-language walkthrough of Vutrisiran Injection — what it treats, how it works, how it’s taken, side effects, warnings and interactions. Tap any section to expand it.
🎯What it's used for▾
Vutrisiran injection (brand name Amvuttra) is approved to treat two distinct complications of transthyretin-mediated amyloidosis (ATTR) — a disease where the TTR protein misfolds and builds up in tissues.
- Amvuttra treats the polyneuropathy (nerve damage) of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults
- Amvuttra treats the cardiomyopathy (heart muscle disease) of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults — with the specific goal of reducing cardiovascular death, cardiovascular hospitalizations, and urgent heart failure visits
⚙️How it works▾
Vutrisiran works through RNA interference (RNAi) — a natural biological process the drug is engineered to harness. It is designed to seek out the liver cells that produce the TTR protein. Once inside, it breaks down the messenger RNA (mRNA) — the genetic instruction — that tells the cell to make TTR. With less mRNA, far less TTR protein is produced, and fewer harmful deposits accumulate in nerves and the heart. In clinical studies, vutrisiran reduced TTR protein levels by about 83% on average.
The drug targets both the mutant form of TTR (responsible for hereditary disease) and the normal, wild-type form (responsible for the sporadic form), making it effective across both types of ATTR amyloidosis.
💊How it's taken▾
Amvuttra is given as a subcutaneous injection (under the skin) by a healthcare professional once every 3 months — one of the least-frequent dosing schedules for any amyloidosis therapy. It is injected into the abdomen, thigh, or upper arm.
- If stored in the refrigerator, allow Amvuttra to warm to room temperature for 30 minutes before use
- Inspect the solution before injection — it should be clear and colorless to yellow; do not use it if it is cloudy, discolored, or contains particles
- If a dose is missed, administer it as soon as possible, then resume the every-3-month schedule from that most recently given dose
🤒Side effects — in detail▾
The most common side effects reported in clinical trials of Amvuttra include:
- Pain in the arms or legs
- Joint pain (arthralgia)
- Shortness of breath (dyspnea)
- Decreased vitamin A blood levels
- Injection site reactions such as bruising, redness, pain, itching, or warmth (these were generally mild and temporary)
A more serious but less common concern is atrioventricular (AV) heart block — a disruption in the heart's electrical system — which was reported in a small number of patients. Tell your doctor right away if you notice slow or irregular heartbeat, dizziness, or fainting. Also seek care promptly if you develop night blindness or other eye symptoms, which can signal vitamin A deficiency.
⚠️Warnings & precautions▾
- Reduced vitamin A levels: Amvuttra reliably lowers vitamin A in the blood (by roughly 62–65%). All patients should take a daily vitamin A supplement at the recommended daily allowance while on this medication. Taking higher doses will not safely correct blood levels and is not recommended. If you develop eye symptoms — especially difficulty seeing in low light (night blindness) — see an ophthalmologist promptly.
- Heart rhythm changes: Cases of atrioventricular (AV) block, including one case of complete heart block, were reported in the polyneuropathy clinical trial. Report any new heart palpitations, slow heartbeat, dizziness, or fainting to your doctor immediately.
🔀What it interacts with▾
Based on laboratory studies, vutrisiran is not expected to cause meaningful drug interactions. Here is what the evidence shows:
- Vutrisiran does not appear to inhibit or be broken down by cytochrome P450 (CYP) liver enzymes — the main pathway through which many common drugs interact
- It is not expected to affect drug transporter proteins in the body
- No clinical drug-drug interaction studies have been conducted with vutrisiran in humans, so tell your doctor and pharmacist about all medications, supplements, and vitamins you take — especially since vitamin A supplementation is recommended during treatment
This is not a complete list of interactions. Always talk with your doctor or pharmacist before starting, stopping, or changing any medication.
📋Before taking it▾
- Pregnancy: It is not known whether Amvuttra is safe during human pregnancy. Animal studies showed potential harm to developing fetuses at doses associated with maternal toxicity. Because Amvuttra also lowers vitamin A — which is essential for normal fetal development — the risks are uncertain. Discuss your pregnancy plans with your doctor.
- Breastfeeding: It is unknown whether vutrisiran passes into breast milk or affects a nursing infant. Weigh the benefits of breastfeeding against any potential risk with your doctor.
- Children: Safety and effectiveness in pediatric patients have not been established.
- Older adults (65+): No dose adjustment is needed. Older adults were well-represented in clinical trials and showed similar safety and effectiveness to younger patients.
- Kidney disease: No dose adjustment is needed for mild or moderate kidney impairment. Amvuttra has not been studied in severe kidney disease or end-stage renal disease.
- Liver disease: No dose adjustment is needed for mild or moderate liver impairment. Amvuttra has not been studied in severe liver disease or in patients who have had a liver transplant.
- Vitamin A supplementation: All patients on Amvuttra should take a daily vitamin A supplement at the recommended daily allowance — this is a standard part of treatment.
📡Pharmacodynamics — effects in the body▾
Amvuttra consistently lowers TTR protein levels in the blood — by an average of about 83% in patients with the polyneuropathy form of the disease, with similar reductions seen across different genetic mutations, ages, sexes, and races. In patients with heart muscle disease (ATTR-CM), the degree of TTR reduction was comparable. Heart failure biomarkers (NT-proBNP and Troponin I) also favored Amvuttra over placebo. As a notable trade-off, vitamin A (retinol) levels in the blood fall substantially — by about 62–65% — because TTR normally serves as a carrier protein for vitamin A; this is why supplementation is recommended.
🔬Pharmacokinetics — how your body processes it▾
After a subcutaneous injection, vutrisiran reaches its peak blood concentration in about 4 hours, then is rapidly cleared from the bloodstream, with a half-life of roughly 5 hours. Despite this short time in the blood, its effects in the liver — where it silences TTR gene expression — last for months, which is why a single injection every 3 months is sufficient. Vutrisiran is broken down inside the liver by natural enzymes (endo- and exonucleases) into small fragments, and about 19% of the unchanged drug is eliminated through the urine. It does not build up in the blood with repeated dosing.
📦How to store it▾
Store at 2°C to 30°C (36°F to 86°F) in the original carton, until ready for use. Do not freeze.
📄 Written from Vutrisiran Injection’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Every point is grounded in that labeling — nothing is invented. How we use AI →
🩺 Pharmacist Counseling Corner
Quick, plain-English answers to the questions patients ask most about Vutrisiran Injection — the kind of thing our pharmacy team would talk through with you at the counter.
What exactly is Amvuttra treating — is it a cure? ▾
How often do I need to come in for my injection? ▾
I heard this medication affects my vitamin A levels — should I be worried? ▾
What side effects should I expect, and what would make me call my doctor? ▾
Is it safe to take my other medications alongside Amvuttra? ▾
I'm older — is Amvuttra safe for me? ▾
Is Vutrisiran Injection available over the counter? ▾
When was Vutrisiran Injection first available? ▾
💬 Answers drafted from Vutrisiran Injection’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Grounded in that labeling — nothing is made up. How we use AI →
🧰 Keep exploring
💊 How Vutrisiran Injection comes
Vutrisiran Injection is available in 1 form. Different forms and brands can have different approved uses, doses, schedules, and directions. Follow the instructions for your exact product, and do not switch forms without guidance from your prescriber or pharmacist.
Injection
How this form is used
- Amvuttra is used to treat the polyneuropathy (nerve damage) of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults
- Amvuttra is used to treat the cardiomyopathy (heart muscle disease) of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults, to reduce cardiovascular death, hospitalizations, and urgent heart failure visits
- Amvuttra is given as a subcutaneous (under-the-skin) injection by a healthcare professional only
- Amvuttra is injected into the abdomen, thigh, or upper arm — avoid the area around the navel, and avoid skin that is scarred, red, inflamed, or swollen
- Amvuttra should be allowed to warm to room temperature for 30 minutes before use if it has been refrigerated
- Amvuttra is given once every 3 months; if a dose is missed, give it as soon as possible and then resume the 3-month schedule from that date
📊 Vutrisiran Injection across the U.S. market
How Vutrisiran Injection appears in the FDA’s National Drug Code Directory — including its dosage forms, strengths, brand names, manufacturers, and FDA-listed product records. These are directory records, not sales or prescription volume.
🔍 Compare Vutrisiran Injection products
A representative set of FDA-listed product records for Vutrisiran Injection — across manufacturers, strengths, and dosage forms, grouped by form with each product’s manufacturer and how the FDA approved it. The same medicine may be made and packaged by different companies, so a single drug can have many directory entries. (It’s also why a refill can look different — a new shape, color, or box — even though it contains the same medication.)
| Strength | Route | Manufacturer / Labeler | Category ⓘ | Details |
|---|---|---|---|---|
| 💊Injection | ||||
| 25 mg/0.5 mL | Subcutaneous | Alnylam Pharmaceuticals, Inc. × 3 listings | NDA | View NDC → |
Showing 3 of 3 products — FDA National Drug Code Directory. See every product, package size and price: browse all 3 Vutrisiran Injection NDCs →
📈 What people report — real-world data (FDA FAERS)
After a medicine reaches the market, patients, doctors and drugmakers can send the FDA reports of problems they think may be linked to it. Here’s what’s been reported for Vutrisiran Injection — a signal of what to watch for, not proof the drug caused it.
Source: openFDA, from the FDA Adverse Event Reporting System (FAERS). These are voluntary reports — they don’t prove the drug caused the effect, and counts reflect how often something was reported, not how often it actually happens. Reports also often name the very problem the medicine is taken for — a common reason to file one is that the drug didn’t help — so for a pain reliever you may see “pain” high on the list; that points to why the report was filed, not to the drug causing the symptom. This counts every report that lists Vutrisiran Injection in any role, so the total runs higher than the FDA’s official dashboard, which counts only cases where the drug is the suspect. For the authoritative figures, see the FDA FAERS Public Dashboard. Always talk to your pharmacist or doctor.
🏛️ The road to your pharmacy
How Vutrisiran Injection went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
🚨 Recall history
A recall is when a specific batch of a medicine is pulled from the market — usually a manufacturing issue, not a problem with the drug itself. Class I is most serious, Class III least.
Source: openFDA drug enforcement (recall) reports.
RxNorm Concept Web — From Ingredient to Every Form and Strength
This page starts with one ingredient concept (IN). The branches below show its dose-form concepts (SCDF), then the available clinical drug and strength concepts (SCD). Combination products remain separate concepts with their own pages.
RxCUI 2604578 1 dose form · 1 strength
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Dose form (SCDF) Prefilled Syringe RxCUI 2604787No current FDA listingClinical drug / strength (SCD) 0.5 ML vutrisiran 50 MG/MLRxCUI 2604788
Look up RxCUI 2604578 in the RxCUI Atlas →
RxNorm is the U.S. National Library of Medicine’s normalized drug vocabulary. Its concept hierarchy can include forms that are not currently marketed; the status on each branch compares that concept with current FDA listings. RxCUIs identify vocabulary concepts, not individual packages or manufacturers.
📦 Supply & shortage status
Whether Vutrisiran Injection is in short supply in the U.S. right now, plus any shortage history the FDA has on record. A shortage usually reflects a manufacturing or demand issue — not a safety problem with the drug.
Source: openFDA, from the FDA Drug Shortages database.
🏷️ Sold under these brand names
🏭 Manufacturers & labelers
RxNorm drug class
Vutrisiran Injection belongs to the Small Interfering RNA class.
Classified by RxNorm RxClass — the U.S. National Library of Medicine's standardized drug-classification service (Established Pharmacologic Class from the FDA, the ATC drug family from the WHO, and mechanism of action). Reference only, not medical advice.
🔬 Where this comes from
Core label, product, RxNorm, safety, and utilization data on this page come from the sources identified below. AI-written, editorial, and licensed sections are labeled separately.
How we combine label and product data
HelloPharmacist combines public FDA and NLM records; we don’t author the underlying clinical facts. We identified 1 FDA-filed SPL label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. The representative label is the starting point for the displayed reference monograph; the 12 largest stored in-scope SPL records, which may include that representative, are compared section by section, and the longest available version of each section is retained. Forms, routes, brands, labelers, product-listed strengths, and product-record counts are aggregated separately from ingredient-matched FDA NDC Directory records; package records listed above are associated with the linked SPL; and the normalized concept hierarchy comes from NLM RxNorm.
Where a section is identified as AI-written, its plain-language text is generated from bounded label evidence. New draft drug pages remain in the admin review queue before first publication. Alnylam Pharmaceuticals, Inc. is the representative DailyMed label linked for verification and dates; it is not the sole source used for the page.
This is general education, not medical advice — always consult your doctor or pharmacist about your medications and any medical condition. For the exact wording of the linked representative label, view that label on DailyMed; some displayed composite sections may come from other in-scope SPL labels. Learn how we use AI and our data sources & update cadence.