With colleagues, we showed that Setmelanotide is effective in treating severe obesity caused by POMC or LEPR deficiency
Working with Rhythm Pharmaceuticals and many clinical collaborators worldwide, we showed in Phase 3 clinical trials that the drug setmelanotide is a safe and effective treatment for obesity caused by POMC or LEPR deficiency.
The Melanocortin 4 Receptor (MC4R), plays a critical part in bodyweight regulation. Setmelanotide is an MC4R agonist and in this study was trialled in several countries in patients with severe obesity due to either pro-opiomelanocortin (POMC) deficiency or leptin receptor (LEPR) deficiency. Participants aged 6 years or older received open-label setmelanotide. After approximately 1 year, eight (80%) participants in the POMC trial and five (45%) participants in the LEPR trial achieved at least 10% weight loss. This degree of weight loss led to significant clinical benefits for patients who find it very hard to lose weight through other means. Ongoing trials will test whether Setmelanotide is effective in other genetic obesity syndromes affecting this pathway.