An injection to prevent attacks of acute hepatic porphyria
Givosiran
An under-the-skin injection used to reduce attacks in acute hepatic porphyria, a group of rare inherited conditions.
What is Givosiran?
Givosiran is a specialist medicine used to reduce attacks in people with acute hepatic porphyria, a group of rare inherited conditions that cause episodes of severe pain and other symptoms. It is a type of RNA interference (siRNA) therapy that lowers the build-up of harmful substances in the liver that trigger attacks. It is given as an injection under the skin on a regular schedule. The most common side effects are reactions where the injection is given, and the team keeps an eye on kidney function and liver blood tests during treatment.
Education and reference only. This is a plain-language guide to Givosiran — 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
Givosiran is a medicine used to lower the frequency of attacks in people with acute hepatic porphyria, a group of rare inherited conditions in which the liver produces too much of certain harmful substances that build up and trigger attacks of severe abdominal pain and other symptoms. It is a type of RNA interference (siRNA) therapy, a newer kind of medicine that reduces the production of a specific protein. It is given as an injection under the skin on a regular schedule and is prescribed and supervised by a specialist porphyria service.
How it works
Attacks of acute hepatic porphyria are driven by a build-up of harmful intermediate substances when the liver is making haem, the iron-containing part of blood. Givosiran works by quietening down a particular instruction in liver cells, so the liver makes less of an early enzyme in this pathway. With this enzyme reduced, fewer of the harmful substances build up, which lowers how often attacks happen. Because the effect needs maintaining, it is given on a regular schedule rather than only during an attack.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist injection used in the UK to reduce attacks in people with acute hepatic porphyria.
Practical use
How to take Givosiran
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Have the injection under the skin on the regular schedule your specialist team arranges.
- Attend for the blood tests your team asks for, as kidney function and liver blood tests are monitored.
- Keep avoiding known porphyria triggers as advised, alongside this treatment.
- Tell your team about any reactions where the injection is given, or new nausea or tiredness.
- Continue treatment as planned, as it works by preventing attacks over time.
Weighing it up
Advantages & disadvantages of Givosiran
Advantages
- Reduces how often attacks of acute hepatic porphyria happen.
- Tackles the build-up of the substances that trigger attacks, at their source.
- Given as an injection under the skin on a regular schedule.
Disadvantages
- Commonly causes reactions where the injection is given, and can cause nausea and tiredness.
- Can affect kidney function and liver blood tests, so monitoring is needed.
- Prevents attacks over time rather than treating an attack in progress.
Practical use
Good to know
The key point with givosiran is that it is used to prevent attacks over time, by reducing the build-up of the substances that trigger them, rather than to treat an attack that is already happening. It is given as an injection under the skin on a regular schedule set by the specialist team. The most common side effects are reactions where the injection is given, such as redness or pain, along with nausea and tiredness in some people. Two things the team keeps an eye on are kidney function and liver blood tests, as these can change during treatment, so regular blood tests are part of the plan. It is used alongside the general measures that help with porphyria, such as avoiding known triggers. The team reviews how well it is working by looking at how often attacks occur.
Who should not take it / use with caution
- People who have had a serious allergic reaction to givosiran should not use it.
- It is used with care, and with monitoring, in people with kidney or liver problems.
- It is used with care, and under specialist guidance, in pregnancy.
- It should only be used under a specialist porphyria service.
Monitoring
- Regular blood tests to check kidney function during treatment.
- Checking liver blood tests over time.
- Reviewing how often attacks happen to judge how well it is working.
Side effects
- Redness, pain or swelling where the injection is given.
- Nausea, tiredness or a rash in some people.
- Changes in kidney function or liver blood tests, which monitoring is designed to catch.
Key interactions
- It can affect how the liver handles some other medicines, so tell your team about everything you take.
- There are few other well-established routine interactions, but always give a full medicines list.
- It is used alongside, not instead of, avoiding known porphyria triggers.
Available as: A solution for injection under the skin, given by a healthcare professional.
Answers
Givosiran: frequently asked questions
What is givosiran used for?
It is used to reduce how often attacks happen in people with acute hepatic porphyria, by lowering the build-up of the substances that trigger attacks.
Does it treat an attack that is happening?
It is used to prevent attacks over time rather than to treat an attack in progress, which is managed in other ways.
How is it given?
It is given as an injection under the skin on a regular schedule arranged by your specialist porphyria service.
What does my team monitor?
Your team keeps an eye on your kidney function and liver blood tests during treatment, so regular blood tests are part of the plan.
What are the most common side effects?
Reactions where the injection is given are the most common, along with nausea and tiredness in some people.
Authoritative sources
- BNF
- NICE CKS
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