An injection for hereditary ATTR amyloidosis nerve disease
Inotersen
An under-the-skin injection used for the nerve disease caused by hereditary transthyretin amyloidosis.
What is Inotersen?
Inotersen 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 antisense oligonucleotide that lowers production of this protein. It is given as an injection under the skin on a regular schedule. It needs close monitoring because it can lower the platelets in the blood and can affect the kidneys, so regular blood and urine tests are essential.
Education and reference only. This is a plain-language guide to Inotersen — 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
Inotersen 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. Inotersen is a type of antisense oligonucleotide, a medicine that switches down the production of a specific protein. It is given as an injection under the skin on a regular schedule and needs careful monitoring, so it 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. Inotersen works by attaching to the genetic message for transthyretin and switching down its production, so the body makes much less of the protein. With less protein being made, there is less to build up, which helps slow the nerve damage. It is given by injection under the skin on a regular schedule to keep this effect going.
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 Inotersen
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Have or give the injection under the skin on the regular schedule your specialist team arranges.
- Attend for the regular blood and urine tests, which check your platelets and kidneys.
- Report unusual bruising, bleeding or tiny red spots on the skin straight away, as these can signal low platelets.
- Tell your team about reduced or frothy urine, swelling or other possible kidney signs.
- Take any vitamin A supplement you are advised, as the medicine can lower vitamin A levels.
Weighing it up
Advantages & disadvantages of Inotersen
Advantages
- Slows the nerve disease caused by hereditary transthyretin amyloidosis.
- Reduces production of the abnormal protein that builds up and damages nerves.
- Given as an injection under the skin rather than as a drip into a vein.
Disadvantages
- Can lower the platelets in the blood, sometimes severely, raising the risk of bleeding.
- Can affect the kidneys, so regular blood and urine tests are essential.
- Requires frequent monitoring and specialist supervision.
Practical use
Good to know
The most important thing to understand about inotersen is that it needs close, regular monitoring for two specific reasons. First, it can lower the platelets in the blood, the cells that help blood clot, sometimes severely, which raises the risk of bruising and bleeding; regular blood tests check the platelet count, and any unusual bruising, bleeding or tiny red spots on the skin should be reported straight away. Second, it can affect the kidneys, including a kind of inflammation called glomerulonephritis, so urine and kidney blood tests are checked regularly too. Because of these risks, treatment is only continued while the monitoring stays acceptable. People are usually advised about the signs to watch for between appointments. It also lowers vitamin A levels, so a supplement may be advised. It slows the nerve disease rather than curing the condition.
Who should not take it / use with caution
- People who have had a serious allergic reaction to inotersen should not use it.
- It is not suitable for people with low platelet counts or certain kidney problems.
- It is used with care, and under specialist guidance, in pregnancy.
- It should only be used under a specialist amyloidosis service with regular monitoring in place.
Monitoring
- Regular blood tests to check the platelet count, because of the risk of low platelets.
- Regular urine and blood tests to check kidney function.
- Reviewing nerve symptoms and overall function over time, and vitamin A status.
Side effects
- A drop in platelets, which can cause bruising or bleeding and is checked with regular blood tests.
- Kidney problems, including inflammation, checked with urine and blood tests.
- Reactions where the injection is given, and flu-like symptoms in some people.
Key interactions
- It is used with extra care alongside medicines that thin the blood or affect platelets, because of the bleeding risk.
- Medicines that can affect the kidneys are reviewed when taking inotersen.
- Tell your team about everything you take, including over-the-counter painkillers and supplements.
Available as: A solution for injection under the skin.
Answers
Inotersen: frequently asked questions
What is inotersen 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.
Why do I need regular blood tests?
Inotersen can lower the platelets in your blood, sometimes severely, so regular blood tests check the count to keep you safe.
What should I report between appointments?
Report unusual bruising, bleeding or tiny red spots on the skin, and any reduced or frothy urine or swelling, as these can signal low platelets or kidney problems.
How is it given?
It is given as an injection under the skin on a regular schedule arranged by your specialist amyloidosis service.
Does it affect the kidneys?
It can affect the kidneys, including a kind of inflammation, so urine and kidney blood tests are checked regularly during treatment.
Authoritative sources
- BNF
- NICE CKS
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