An RNA-interference (siRNA) injection
Nedosiran
An RNA-interference injection used to reduce oxalate levels in primary hyperoxaluria type 1 and help protect the kidneys.
What is Nedosiran?
Nedosiran is a specialist medicine for the rare inherited condition primary hyperoxaluria type 1, in which the body makes too much of a substance called oxalate that can build up and damage the kidneys. It is a type of RNA-interference (siRNA) treatment, given as an injection under the skin, that lowers the body's production of oxalate to help protect the kidneys. The most common side effect is a reaction where the injection is given. It is prescribed and supervised by a specialist team.
Education and reference only. This is a plain-language guide to Nedosiran — 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
Nedosiran is a medicine for primary hyperoxaluria type 1, a rare inherited condition in which the liver overproduces a substance called oxalate. Too much oxalate can form crystals and stones and gradually damage the kidneys. Nedosiran is a type of treatment known as RNA interference, or siRNA, which works by reducing how much oxalate the body makes. It is given as an injection under the skin and is used as a long-term treatment under the care of a specialist team familiar with this rare condition.
How it works
In primary hyperoxaluria type 1, the liver makes too much oxalate because of a faulty gene. Nedosiran is an RNA-interference (siRNA) medicine that lowers the activity of an enzyme involved in producing oxalate, so the body makes less of it. With less oxalate being produced, there is less to build up in the kidneys and urinary tract, which helps protect the kidneys and reduce the formation of crystals and stones. Because it works steadily over time, it is given as a regular injection rather than as a one-off.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist injection used to lower oxalate in people with the rare inherited condition primary hyperoxaluria type 1.
Practical use
How to take Nedosiran
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Use it on the regular schedule your specialist team sets, injecting it under the skin as you have been trained.
- Inject it at the recommended intervals rather than waiting for symptoms, as it works over time.
- Continue other measures you are advised, such as drinking plenty of fluids, alongside it.
- Report any troublesome or persistent reactions where the injection is given.
- Attend your monitoring appointments to check oxalate levels and kidney health.
Weighing it up
Advantages & disadvantages of Nedosiran
Advantages
- Lowers the body's production of oxalate to help protect the kidneys in primary hyperoxaluria type 1.
- Given as an injection under the skin, which can be done at home after training.
- Works steadily over time as a long-term treatment for a rare condition.
Disadvantages
- Commonly causes reactions where the injection is given.
- Needs regular injections and ongoing specialist monitoring.
- Treats only one part of a complex condition, alongside fluids and other measures.
Practical use
Good to know
Nedosiran is a long-term treatment for a lifelong, rare condition, so taking it regularly as scheduled is important to keep oxalate levels lower over time. It is given as an injection under the skin, often by the person themselves or a carer after training, which makes treatment at home possible. The most common side effect is a reaction where the injection is given, such as redness, pain or swelling, which usually settles on its own. It is one part of overall care for primary hyperoxaluria, which also usually includes drinking plenty of fluids and other measures advised by the specialist team. Because the condition is rare and complex, treatment is overseen by specialists who monitor oxalate levels and kidney health.
Who should not take it / use with caution
- People who have had a serious allergic reaction to nedosiran should not use it.
- It is used under specialist guidance, with care in pregnancy where benefits and risks are weighed.
- It should only be used under a specialist service experienced in primary hyperoxaluria.
Monitoring
- Checking oxalate levels to see how well treatment is working.
- Monitoring kidney function and overall kidney health over time.
- Checking the person is confident with the injection technique and schedule.
Side effects
- Redness, pain or swelling where the injection is given, usually settling on its own.
- Other reactions at the injection site in some people.
- Rarely, allergic-type reactions, which should be reported to the specialist team.
Key interactions
- There are few well-established routine medicine interactions, but tell your team about all your medicines.
- It is used alongside, not instead of, other measures such as good fluid intake.
- Your team will take your full treatment plan into account when prescribing it.
Available as: A solution for injection under the skin, often given by the patient or a carer.
Answers
Nedosiran: frequently asked questions
What is nedosiran used for?
It is used to treat the rare inherited condition primary hyperoxaluria type 1 by lowering the body's production of oxalate to help protect the kidneys.
How does it work?
It is an RNA-interference (siRNA) medicine that reduces the activity of an enzyme involved in making oxalate, so the body produces less of it.
How is it given?
It is given as an injection under the skin on a regular schedule, often by the person themselves or a carer after training.
What is the most common side effect?
The most common side effect is a reaction where the injection is given, such as redness, pain or swelling, which usually settles on its own.
Is it the only treatment I need?
No. It is one part of overall care, which usually also includes drinking plenty of fluids and other measures advised by your specialist team.
Authoritative sources
- BNF
- NICE CKS
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