vutrisiran sodium at a glance
Medicine class
Transthyretin-directed small interfering RNA therapy
Licensed uses
Selected transthyretin amyloidosis affecting peripheral nerves or the heart in adults
Administration
Injection under the skin using a ready-to-use, single-use pre-filled syringe
Important precaution
It lowers blood vitamin A; prescribed supplementation and assessment of new eye symptoms are important
What is vutrisiran sodium used for?
UK-licensed uses in adults are:
Hereditary transthyretin amyloidosis with stage 1 or stage 2 polyneuropathy.
Wild-type or hereditary transthyretin amyloidosis with cardiomyopathy.
How does vutrisiran sodium work?
Vutrisiran is delivered mainly to liver cells, where it uses RNA interference to break down messenger RNA carrying instructions for transthyretin production. This lowers both variant and normal transthyretin in the blood, reducing the protein available to form amyloid deposits.
vutrisiran sodium preparations and strengths
Injection
Route: Parenteral
Strengths: 25 mg
How to take vutrisiran sodium
It is injected under the skin by a healthcare professional or, after training, by the patient or a caregiver.
Suitable sites are the abdomen, thigh or upper arm; another person must give an upper-arm injection.
Avoid scarred, tender, red, swollen, inflamed or hardened skin and the area immediately around the navel.
Inspect the solution before use and do not use it if cloudy, discoloured or containing particles.
If stored cold, leave the carton at room temperature for about 30 minutes; do not use another warming method.
Use each pre-filled syringe once and dispose of it in a sharps container.
vutrisiran sodium side effects
Common or expected effects
- Injection-site bruising, redness, pain, itching or warmth; these reactions were generally mild and temporary in studies.
- Raised alanine transaminase on blood testing.
- Raised alkaline phosphatase on blood testing.
Get urgent medical advice
- New night-vision difficulty, persistent dry eyes, cloudy vision or other eye symptoms may indicate vitamin A deficiency and need prompt clinical assessment.
- Symptoms of a severe allergic reaction require emergency help.
vutrisiran sodium in pregnancy
Vutrisiran should not be used during pregnancy because altered vitamin A levels may harm fetal development and human pregnancy data are unavailable. Pregnancy should be excluded before treatment and effective contraception used. Anyone planning or discovering a pregnancy should contact the specialist promptly and should not independently change prescribed vitamin A supplementation.
vutrisiran sodium while breastfeeding
It is unknown whether vutrisiran passes into human milk. The specialist and patient should decide between breastfeeding and treatment after weighing the benefits for the child and the woman.
vutrisiran sodium interactions
Formal clinical interaction studies have not been performed, but clinically significant medicine interactions are not expected from vutrisiran's metabolic properties.
Vitamin A supplements
Supplementation is usually prescribed because vutrisiran lowers vitamin A; taking more than advised may be harmful, particularly around pregnancy.
Cytochrome P450 inhibitors or inducers
These are not expected to meaningfully alter vutrisiran, and vutrisiran is not expected to cause clinically significant CYP-mediated interactions.
Common questions about vutrisiran sodium
Answers are fully visible for fast scanning and source review.
Is vutrisiran sodium the same as Amvuttra?
Vutrisiran sodium is the active ingredient; Amvuttra is the brand name of the selected UK product.
What type of treatment is vutrisiran sodium?
It is a liver-targeted small interfering RNA treatment that switches down production of transthyretin protein.
Which types of amyloidosis can vutrisiran sodium treat?
Its UK licence covers hereditary ATTR amyloidosis with stage 1 or 2 polyneuropathy and wild-type or hereditary ATTR amyloidosis with cardiomyopathy in adults.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.