1.1 Metreleptin is recommended, within its marketing authorisation, as an option for treating the complications of leptin deficiency in lipodystrophy for people who are 2 years and over and have generalised lipodystrophy.
1.2 Metreleptin is recommended as an option for treating the complications of leptin deficiency in lipodystrophy for people who are 12 years and over, have partial lipodystrophy, and do not have adequate metabolic control despite having standard treatments. It is only recommended if they have an HbA1c level above 58 mmol/mol (7.5%), or fasting triglycerides above 5.0 mmol/litre, or both.
1.3 This recommendation is not intended to affect treatment with metreleptin that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. This decision should be made jointly by the clinician, the child or young person and their parents or carers.
Why the committee made these recommendations
Lipodystrophy is a rare and serious condition that severely affects the quality of life of people with the condition, and their families and carers. For children and young people with the condition, it may also shorten their life expectancy. Supportive care includes lifestyle modifications such as an extreme low-fat diet and exercise, cosmetic surgery, and medication for diabetes and to lower lipids.
There is no trial directly comparing metreleptin with supportive care. Results from clinical studies suggest that metreleptin appears to improve hyperphagia and reduces HbA1c and triglyceride levels in people with lipodystrophy. An indirect comparison with supportive care also suggests that metreleptin is more effective at improving HbA1c, triglyceride and liver enzyme levels, as well as episodes of acute pancreatitis at 12-month follow up. However, metreleptin's long-term effect and several assumptions in the economic modelling are uncertain. Despite this, metreleptin is likely to provide important clinical benefit and improve quality of life for people who have lipodystrophy, parents and carers. It also provides value for money within the context of a highly specialised service. So, it is recommended for use in the NHS.