hrp0095p1-472 | Fat, Metabolism and Obesity | ESPE2022

Venture: Design of a Phase 3 Multicenter, 1-Year, Open-Label Trial of Setmelanotide in Pediatric Patients Aged 2 to <6 Years With Rare Genetic Diseases of Obesity

Farooqi Sadaf , Mohamed Iqbal Anoop , Fennoy Ilene , M. Kelsey Megan , F. Verge Charles , Cokkinias Casey , Lee Hak-Myung , Navarria Andrea , Argente Jesús

Background: Rare genetic diseases of obesity are often driven by gene variants in the melanocortin-4 receptor (MC4R) pathway. The MC4R agonist setmelanotide demonstrated significant reductions in body weight in patients ≥6 years old with various rare genetic diseases of obesity, including proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency and Bardet-Biedl syndrome (BBS). While these condit...