National Institute for Health and Care Excellence (NICE) has published draft guidance which recommends metreleptin for use within the NHS for treating complications of leptin deficiency in lipodystrophy in patients aged two years and over who have generalised lipodystrophy.
Metreleptin is further recommended as an option for patients aged 12 years and over who have partial lipodystrophy and do not have adequate metabolic control despite having standard treatments. It is only recommended if the patient’s HbA1c level is above 7.5 per cent and/or fasting triglycerides are above 5.0 mmol/L.
The draft guidance is a u-turn on a previous decision issued in 2019 which did not recommend the treatment. Following an appeal by the manufacturer and others, NICE agreed to retract that guidance and postpone evaluation of metreleptin.
The agency says this new positive recommendation follows the receipt of additional information and extensive liaison with the company.
The draft guidance concludes that metreleptin (also called Myalepta) is likely to provide important clinical benefit and improve quality of life for people with lipodystrophy, while providing value for money within the context of a highly specialised service.
There is no trial directly comparing metreleptin with standard 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 standard care also suggests that metreleptin is more effective at improving HbA1c, triglyceride and liver enzyme levels at 12-month follow-up.
Consultees, including the company, health care professionals and patient/carer organisations, now have the opportunity to appeal against the draft recommendations. If no appeals are received, final guidance is expected to be published next month.