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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...

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hrp0098p1-296 | Late Breaking 1 | ESPE2024

Evaluating Setmelanotide Treatment for 12 Months in Pediatric Age Groups With Rare Melanocortin-4 Receptor Pathway–Related Obesity: Efficacy in Weight Reduction and Safety Outcomes

Kühnen Peter , L. T. van den Akker Erica , H. Shoemaker Ashley , Okorie Uzoma , F. Verge Charles , Fennoy Ilene , M. Kelsey Megan , M. Haqq Andrea , L. Roth Christian , C. Garrison Jill , Wabitsch Martin , Farooqi Sadaf , Argente Jesús

Objectives: Hyperphagia and severe obesity may result from impaired melanocortin-4 receptor (MC4R) signaling due to rare biallelic variants in POMC or PCSK1 (proopiomelanocortin [POMC] deficiency) or LEPR (leptin receptor [LEPR] deficiency), Bardet-Biedl syndrome (BBS) or acquired hypothalamic obesity (HO). Previously, setmelanotide in patients aged 2-17 years was well tolerated and improved weight-related measures and hunger severit...

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ESPE Abstracts

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

Evaluating Setmelanotide Treatment for 12 Months in Pediatric Age Groups With Rare Melanocortin-4 Receptor Pathway–Related Obesity: Efficacy in Weight Reduction and Safety Outcomes

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