A replacement for the hormone leptin in lipodystrophy
Metreleptin
A daily under-the-skin injection that replaces the hormone leptin in people with lipodystrophy.
What is Metreleptin?
Metreleptin is a specialist medicine used to treat lipodystrophy, a rare condition in which the body lacks normal fat tissue and, as a result, is short of the hormone leptin. Leptin helps control appetite and how the body handles sugar and fat, so a lack of it leads to problems such as high blood sugar and high blood fats. Metreleptin replaces the missing leptin and is given as a daily injection under the skin. It can lower blood sugar, so doses of diabetes medicines, especially insulin, may need reducing to avoid hypoglycaemia. The body can sometimes make antibodies against it that reduce how well it works, and there is a recognised but rare signal of a blood cancer called lymphoma, so it is closely supervised by a specialist team.
Education and reference only. This is a plain-language guide to Metreleptin — 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
Metreleptin is a man-made version of leptin, a hormone normally made by the body's fat tissue. In lipodystrophy, the body has too little fat tissue and therefore too little leptin, which disturbs appetite and the way the body handles sugar and fat, often causing high blood sugar and high levels of fat in the blood. Metreleptin replaces the missing hormone to help bring these under control. It is given as an injection under the skin, usually once a day, often by the person or a carer after training, and is prescribed and monitored by a specialist team experienced in this rare condition.
How it works
Leptin acts as a signal that tells the brain about the body's energy stores, helping to control appetite and to keep blood sugar and blood fats in balance. In lipodystrophy this signal is missing, so the body behaves as if it is starving even when it is not, driving up appetite, blood sugar and fat levels. By replacing leptin, metreleptin restores this signal, which can reduce appetite, lower blood sugar and improve blood fats over time. Because it works as ongoing hormone replacement, it is given every day, and its effects on sugar mean diabetes treatments often need adjusting.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist medicine used in the UK to treat the complications of lipodystrophy, a rare condition where the body lacks normal fat tissue and the hormone leptin.
Practical use
How to take Metreleptin
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Inject it under the skin once a day, at about the same time, as your specialist team has trained you.
- Rotate the injection site and follow the instructions for preparing and storing the medicine.
- Watch for signs of low blood sugar, especially when starting, as doses of diabetes medicines such as insulin may need reducing.
- Tell your team if the medicine seems to be working less well, as the body can make antibodies against it.
- Attend your regular reviews so blood sugar, blood fats and overall health can be checked.
Weighing it up
Advantages & disadvantages of Metreleptin
Advantages
- Replaces the missing hormone leptin, helping control high blood sugar and high blood fats in lipodystrophy.
- Can reduce appetite, which is often raised in this condition.
- Targets the underlying hormone problem in a rare condition with few other options.
Disadvantages
- Given as a daily injection under the skin, which needs training.
- Can cause blood sugar to drop too low when used with diabetes medicines such as insulin.
- The body can make antibodies that reduce how well it works, and there is a rare signal of lymphoma.
Practical use
Good to know
A practical point with metreleptin is that, because it lowers blood sugar, any diabetes medicines, particularly insulin, may need to be reduced when it is started to avoid blood sugar dropping too low, so blood sugar is watched closely. Two important safety points stand out. First, the body can sometimes make antibodies against metreleptin that may reduce how well it works or, rarely, affect the body's own leptin, so loss of effect or new infections are taken seriously. Second, there is a recognised, although rare, signal of a blood cancer called lymphoma in people with this condition, which the specialist team keeps in mind and monitors for. It is given as a daily injection under the skin, and the team will train you to prepare and inject it safely. As with any hormone, careful, regular review is part of treatment.
Who should not take it / use with caution
- People who have had a serious allergic reaction to metreleptin should not use it.
- It is used with particular care in people taking insulin or other diabetes medicines, which often need reducing.
- It is used with care, and under specialist guidance, in pregnancy.
- It should only be used under a specialist team experienced in lipodystrophy, with regular monitoring.
Monitoring
- Regular blood sugar checks, with adjustment of diabetes medicines, especially after starting.
- Blood tests for blood fats and overall response to treatment.
- Watching for loss of effect linked to antibodies, and for any signs that need checks for lymphoma.
Side effects
- Low blood sugar, especially when used with diabetes medicines, with symptoms such as shakiness, sweating or confusion.
- Reactions where the injection is given, such as redness or bruising.
- Headache, tiredness or tummy upset in some people.
- Rarely but importantly, loss of effect linked to antibodies, or signs that should prompt checks for lymphoma.
Key interactions
- It lowers blood sugar, so insulin and other diabetes medicines often need their doses reduced.
- It may change how some other medicines are handled by the body, so tell your team about all your medicines.
- Give a full medicines list to your specialist team so treatment can be adjusted safely.
Available as: A powder made up into a solution for injection under the skin.
Answers
Metreleptin: frequently asked questions
What is metreleptin used for?
It is used to treat the complications of lipodystrophy, a rare condition where the body lacks fat tissue and the hormone leptin, by replacing the missing leptin.
Why might my diabetes medicines need changing?
Metreleptin lowers blood sugar, so insulin and other diabetes medicines often need reducing to avoid blood sugar dropping too low, especially when treatment starts.
Why might it stop working over time?
The body can sometimes make antibodies against metreleptin that reduce how well it works, so tell your team if it seems less effective.
Is there a cancer risk?
There is a recognised but rare signal of a blood cancer called lymphoma in people with this condition, which your specialist team keeps in mind and monitors for.
How is it given?
It is given as an injection under the skin, usually once a day, often by the person or a carer after training from the specialist team.
Authoritative sources
- BNF
- NICE CKS
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