Submitted by aml95 on Wed, 11/12/2024 - 09:58
The Farooqi team recently worked with colleagues across the world to complete a 12 month Phase 3 clinical trial of the Melanocortin 4 receptor agonist, Setmelanotide, in very young children (aged 2-5 years) with severe obesity due to genetic conditions which disrupt the function of the hypothalamic melanocortin pathway. The trial included children with homozygous (bi-allelic) mutations in POMC, PCSK1, LEPR and children with Bardet-Biedl syndrome (BBS) where genes affect the function of cilia. The trial showed 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.
Following publication of the results in the Lancet Diabetes and Endocrinology, the MHRA have extended the license for this medication in the UK, so that children with these disorders can now be treated from the age of 2. In Cambridge, we treat children and adults with these conditions from across the UK following commissioning by NHS England. We are delighted by this development, as effective treatment from a young age limits the physical, mental and social impact of severe obesity on children.
Reference:
Argente J, Verge CF, Okorie U, Fennoy I, Kelsey MM, Cokkinias C, Scimia C, Lee HM, Farooqi IS. Setmelanotide in patients aged 2-5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial. Lancet Diabetes Endocrinol. 2024 Nov 12:S2213-8587(24)00273-0. doi: 10.1016/S2213-8587(24)00273-0. Epub ahead of print. PMID: 39549719.
