Setmelanotide Promising as Early Intervention for Rare Obesity in Young Children: Findings from VENTURE Trial
USA: Research shows that the melanocortin-4 receptor (MC4R) agonist setmelanotide effectively lowers BMI in children with early-onset severe obesity linked to single-gene variants that disrupt hunger regulation, satiety, and energy balance through the MC4R signaling pathway. The findings were published online in The Lancet Diabetes & Endocrinology on November 13, 2024.
Previous research has shown that aetmelanotide reduces hunger and weight in children aged 6 years and older with proopiomelanocortin (POMC) deficiency (including biallelic variants in proprotein convertase subtilisin/kexin type 1), leptin receptor (LEPR) deficiency, or Bardet-Biedl syndrome (BBS), all classified as MC4R pathway diseases. However, no approved therapies currently exist for children under 6 years with these conditions.
Against the above background, Prof Jesús Argente, IMDEA Food Institute, Madrid, Spain, and colleagues aimed to evaluate the efficacy and safety of setmelanotide in patients aged 2–5 years with POMC or LEPR deficiency or Bardet-Biedl syndrome through phase 3, open-label VENTURE trial.
The Phase 3, open-label, multicenter trial was conducted at six sites across the USA, the UK, Spain, and Australia. It included children aged 2–5 years with hyperphagia and obesity caused by biallelic POMC (including PCSK1) LEPR variants or genetically confirmed BBS. Participants received open-label subcutaneous setmelanotide once daily for 52 weeks, starting at a dose of 0.5 mg, which was increased every two weeks by 0.5 mg until the maximum dose based on weight was reached.
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