vutrisiran
Amvuttra · ATC code N07XX18
- Key facts
- Active substance
- vutrisiran
- ATC code
- N07XX18
- Authorisation
- centrally authorised across the EU (EMA)
- Dosage forms
- Amvuttra 25 mg solution for injection in pre-filled syringe
- Marketing authorisation holder
- Alnylam Netherlands B.V.
- Information as of
- 2026-10-28
Key facts
Vutrisiran is a small interfering RNA (siRNA) that lowers liver production of transthyretin; it is used for transthyretin amyloidosis with polyneuropathy or cardiomyopathy (Amvuttra).
What it is used for
- Hereditary transthyretin amyloidosis in adults with stage 1 or stage 2 polyneuropathy (hATTR-PN).
- Wild-type or hereditary transthyretin amyloidosis in adults with cardiomyopathy (ATTR-CM).
Dosing
- Amvuttra: 25 mg by subcutaneous injection once every 3 months; may be given by a healthcare professional, the patient or a caregiver after guidance.
- Vitamin A supplementation of approximately, but not exceeding, 2,500 IU to 3,000 IU per day is advised.
- A missed dose should be given as soon as possible, then every 3 months from the most recent dose.
- No dose adjustment in patients ≥ 65 years, mild or moderate hepatic impairment, or mild to moderate renal impairment (eGFR ≥ 30 to < 90 mL/min/1.73 m²); in severe hepatic or renal impairment only if the benefit outweighs the risk.
Contraindications
- Severe hypersensitivity (e.g. anaphylaxis) to the active substance or any excipient.
Important warnings
- Vitamin A deficiency: serum vitamin A falls; correct low levels before starting and take daily vitamin A supplementation; ophthalmological assessment for eye symptoms such as night blindness.
- In the first 60 days of pregnancy, too high or too low vitamin A levels may increase the risk of foetal malformation; exclude pregnancy before starting and use effective contraception.
- Vitamin A levels may remain reduced for more than 12 months after the last dose.
Side effects
- Common: injection site reactions (bruising, erythema, pain, pruritus, warmth; mild and transient), alanine transaminase increased, blood alkaline phosphatase increased.
- In HELIOS-B, mild ALT increases occurred in 30% of vutrisiran patients versus 24% with placebo.
Interactions
- No clinical interaction studies; clinically significant interactions are not expected.
Pregnancy and breastfeeding
- Should not be used during pregnancy; if pregnancy is planned, stop vutrisiran and vitamin A supplementation and wait for vitamin A levels to normalise.
- Breast-feeding: decide between stopping breast-feeding or therapy based on benefit and risk.
Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.
Common questions – ask Medelix
For patients
Package leaflet, verbatim
This product has no separate US patient information.
Authorisation and brand names by country
Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.
EU – centrally authorised (all member states)
- AmvuttraAlnylam Netherlands B.V. · EMEA/H/C/005852
Source: European Medicines Agency (EMA), product information
Brand names in 3 more European countries
- Iceland
- Amvuttra
- Liechtenstein
- Amvuttra
- Norway
- Amvuttra
EMA – Article 57 product data (EU/EEA), 2026-10-29
Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.
Other substances in the same group
Another question about this substance?
Medelix answers clinical questions from guidelines, studies and product information – every claim with its source.
Sources
- European Medicines Agency. Amvuttra – EPAR, Product Information. 2026-10-28. https://www.ema.europa.eu/en/documents/product-information/amvuttra-epar-product-information_en.pdf
Editorial: Medelix editorial team · last updated 2026-10-28 · How these pages are made · Report an error
Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.