Setmelanotide is in clinical development for the treatment of rare genetic obesity due to leptin receptor deficiency. The leptin receptor is found on the surface of cells in many organs and tissues of the body, including a part of the brain called the hypothalamus, which controls hunger and thirst. The leptin receptor is involved in the control of body weight by increasing energy expenditure and reducing appetite. Individuals with leptin receptor deficiency experience excessive hunger and weight gain.
Setmelanotide is given as a sub-cutaneous (under the skin) injection. It is believed to restore lost activity of the leptin receptor, re-establishing weight and appetite control in patients. If licensed, setmelanotide will offer a treatment option for patients with obesity due to leptin receptor deficiency who currently have few effective therapies available.