Setmelanotide CAS:1294000-61-5

Setmelanotide is primarily used to treat severe obesity associated with genetic disorders such as pro-opiomelanocortin (POMC) deficiency, leptin receptor (LEPR) deficiency, and Bardet-Biedl syndrome (BBS). These disorders disrupt normal appetite regulation mechanisms, leading to excessive weight gain from an early age. As a selective MC4R agonist, setmelanotide modulates the MC4R pathway in the hypothalamus, a key brain region involved in regulating energy balance and appetite. By activating MC4R, setmelanotide helps to reduce hunger and increase satiety, which can lead to significant weight loss in patients with these rare genetic conditions. Clinical trials have demonstrated the efficacy of setmelanotide in significantly reducing body weight and improving metabolic parameters in patients with POMC or LEPR deficiency. Treatment with setmelanotide has shown to decrease hyperphagia (excessive eating) and improve quality of life for individuals affected by these severe forms of genetic obesity. In addition to its approved indications, setmelanotide is also being investigated for its potential in other forms of obesity and related metabolic disorders. Ongoing research aims to expand its therapeutic applications and explore its efficacy in broader patient populations affected by severe obesity with genetic or metabolic origins. Overall, setmelanotide represents a promising advancement in the treatment of rare genetic disorders associated with severe obesity, offering a targeted therapeutic approach to address the underlying causes of excessive weight gain and improve long-term health outcomes for affected individuals.