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
Publication: The Lancet: Diabetes and Endocrinology
Prof Karine Clément, MD, Prof Erica van den Akker, MD, Prof Jesús Argente, MD, Allison Bahm, MD, Prof Wendy K Chung, MD, Hillori Connors, MS, Kathleen De Waele, MD, Prof I Sadaf Farooqi, PhD, Julie Gonneau-Lejeune, MD, Gregory Gordon, MD, Katja Kohlsdorf, MD, Prof Christine Poitou, MD, Lia Puder, MD, James Swain, MD, Murray Stewart, DM, Guojun Yuan, PhD, Prof Martin Wabitsch, MD, Prof Peter Kühnen, MD
30 October 2020
Summary:
In this international phase 3 study, researchers wanted to see if the drug Setmelanotide could help people whose severe obesity is caused by pro-opiomelanocortin (POMC) or leptin (LEPR) deficiency.
Melanocortin 4 Receptor (MC4R), plays a critical part in bodyweight regulation and Setmelanotide is an MC4R agonist. This trial was conducted in several countries with participants with severe obesity due to either POMC deficiency LEPR deficiency.
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. Researchers found Setmelanotide to be a safe and effective treatment for people with POMC or LEPR deficiency.
This research could benefit people who find it hard to lose weight. There are ongoing trials to test whether Setmelanotide is effective in other genetic obesity syndromes.