An injection for hereditary ATTR amyloidosis nerve disease
Vutrisiran
An under-the-skin injection used for the nerve disease caused by hereditary transthyretin amyloidosis.
What is Vutrisiran?
Vutrisiran is a specialist medicine used to treat the nerve disease (polyneuropathy) caused by hereditary transthyretin (hATTR) amyloidosis, a rare inherited condition in which an abnormal protein builds up and damages the nerves. It is a type of RNA interference (siRNA) therapy that lowers production of this protein. It is given as an injection under the skin on a spaced-out schedule, which many people find convenient. People taking it are usually given a vitamin A supplement, as the medicine lowers vitamin A levels.
Education and reference only. This is a plain-language guide to Vutrisiran — it deliberately contains no doses. Doses depend on the person, the brand and the reason for treatment, and belong with your prescriber. Always check the BNF, the product labelling (SmPC) and follow medical advice.
What it is
Vutrisiran is a medicine used to treat the nerve disease, called polyneuropathy, in people with hereditary transthyretin (hATTR) amyloidosis. In this inherited condition, a protein called transthyretin is made in an abnormal form that clumps together and builds up in tissues, including the nerves, causing damage. Vutrisiran is a type of RNA interference (siRNA) therapy that reduces how much transthyretin the body makes. It is given as a quick injection under the skin on a spaced-out schedule and is prescribed and supervised by a specialist service.
How it works
In hereditary transthyretin amyloidosis, the liver makes an abnormal form of the protein transthyretin, which clumps together and is deposited in tissues such as the nerves, causing the nerve disease. Vutrisiran works by quietening down the instruction that tells the liver to make transthyretin, so much less of the protein is produced. With less protein being made, there is less to build up, which helps slow the nerve damage. It is designed so each injection lasts a good while, so it is given on a spaced-out schedule. Because transthyretin also helps carry vitamin A in the blood, lowering it can reduce vitamin A levels, which is why a supplement is given.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist injection used in the UK for nerve disease caused by hereditary transthyretin (hATTR) amyloidosis.
Practical use
How to take Vutrisiran
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Have the injection under the skin on the spaced-out schedule your specialist team arranges.
- Take the vitamin A supplement you are given, as the medicine lowers vitamin A levels.
- Tell your team about any eye or vision problems, as these can relate to vitamin A.
- Attend your appointments so the team can review how well it is working.
- Continue treatment as planned, as it slows the disease over the long term.
Weighing it up
Advantages & disadvantages of Vutrisiran
Advantages
- Slows the nerve disease caused by hereditary transthyretin amyloidosis.
- Given as a quick injection under the skin on a spaced-out schedule.
- Convenient for many people compared with a regular drip into a vein.
Disadvantages
- Commonly causes reactions where the injection is given, and joint or limb pain in some people.
- Lowers vitamin A levels, so a supplement is needed.
- Slows the nerve disease rather than curing the condition, so it is needed long term.
Practical use
Good to know
A practical advantage of vutrisiran is that it is given as a quick injection under the skin on a spaced-out schedule, rather than as a regular drip into a vein, which many people find more convenient. The most common side effects are reactions where the injection is given, along with joint pain or limb pain in some people. As with the related infusion treatment, because vutrisiran lowers transthyretin, which normally helps carry vitamin A in the blood, vitamin A levels fall, so people are usually given a vitamin A supplement and advised to report any eye or vision problems. It slows the nerve disease rather than curing the condition, so it is a long-term treatment. The specialist team checks how well it is working by reviewing nerve symptoms and overall function over time, and keeps the schedule going as planned.
Who should not take it / use with caution
- People who have had a serious allergic reaction to vutrisiran should not use it.
- It is used with care, and under specialist guidance, in pregnancy.
- It should only be used under a specialist amyloidosis service.
Monitoring
- Checking nerve symptoms and overall function over time to judge how well it is working.
- Keeping an eye on vitamin A status and any eye or vision symptoms.
- Watching for reactions where the injection is given.
Side effects
- Redness, pain or swelling where the injection is given.
- Joint pain or limb pain in some people.
- Low vitamin A levels, which is why a supplement is given.
Key interactions
- There are few well-established routine medicine interactions, but tell your team about all your medicines.
- It is given alongside a vitamin A supplement rather than relying on diet alone.
- Always tell your team about supplements and over-the-counter products you take.
Available as: A solution for injection under the skin, given by a healthcare professional.
Answers
Vutrisiran: frequently asked questions
What is vutrisiran used for?
It is used to treat the nerve disease caused by hereditary transthyretin (hATTR) amyloidosis, by reducing production of the abnormal protein that builds up and damages nerves.
How is it given?
It is given as a quick injection under the skin on a spaced-out schedule, which many people find more convenient than a regular drip into a vein.
Why do I need vitamin A?
The medicine lowers transthyretin, which helps carry vitamin A in the blood, so vitamin A levels fall and a supplement is given; report any vision problems.
How is it different from the drip treatment?
Both reduce production of the abnormal protein, but vutrisiran is a quick under-the-skin injection on a spaced-out schedule rather than a regular drip into a vein.
Does it cure the condition?
No. It slows the nerve disease rather than curing the condition, so it is a long-term treatment.
Authoritative sources
- BNF
- NICE CKS
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