Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials
Analysis of two studies found that patients treated with setmelanotide achieved 10% weight loss at approximately 1 year (8/10 pts in POMC trial and 5/11 pts in LEPR trial) suggesting a potential role in the treatment of obesity and hyperphagia caused by POMC or LEPR deficiency.
Source:
The Lancet Diabetes & Endocrinology
SPS commentary:
Setmelanotide is a melanocortin-4 receptor(MC4R) agonist that has been filed in EU for treatment of POMC deficiency obesity and LEPR deficiency obesity.
MC4R is a component of the leptin–melanocortin pathway, which plays a part in bodyweight regulation. POMC & LEPR deficiency obesities are ultra-rare genetic disorders, with patients suffering from insatiable hunger beginning at very young age.