MC4R agonist now licensed for treatment of young children with POMC, PCSK1, LEPR deficiencies
In a paper published in the Lancet Diabetes and Endocrinology, we report results from a 12 month clinical trial of the Melanocortin 4 receptor agonist, Setmelanotide, in children (aged 2-5 years) with severe obesity due to genetic conditions which disrupt the hypothalamic melanocortin pathway. Working with colleagues in Spain, USA and Australia, we studied children with homozygous (bi-allelic) mutations in POMC, PCSK1, LEPR and children with Bardet-Biedl syndrome (BBS). Treatment led to sustained weight loss with minimal adverse effects including skin pigmentation; mean percent change in BMI at week 52 was −26% (SD 11) in children with POMC or LEPR deficiency with a significant reduction in hunger scores.
Setmelanotide is a MC4R agonist which is currently licensed in the UK and other countries for the treatment of POMC, PCSK1, LEPR deficiencies and BBS, for children (aged 6 years and above) and adults. In Cambridge, we treat patients from across the UK following commissioning by NHS England. As a result of this trial, the MHRA have extended the license so that children age 2 and above can be treated. This is important as effective treatment from a young age limits the physical, mental and social impact of severe obesity in children. We look forward to working with colleagues across the UK to treat children with these conditions.
