The U.S. Food and Drug Administration has approved a supplemental indication for setmelanotide (Imcivree) for chronic weight management in patients ≥6 years of age with obesity due to Bardet-Biedl Syndrome (BBS).

BBS is a rare genetic disease that impairs the hunger signal along the MC4R pathway in the brain, causing insatiable hunger. In addition to insatiable hunger, BBS  is associated with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.

Setmelanotide is a MC4R agonist, targeting the root cause of BBS. It was approved by the FDA in November 2020 for chronic weight management in patients ≥6 years of age with obesity due to the following genetic conditions which cause severe obesity that begins at an early age: proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. Setmelanotide is the first targeted therapy approved for BBS.

The FDA approval of the extended indication was based largely on positive results of a phase 3 clinical trial which enrolled 44 patients with a clinical diagnosis of BBS and obesity. After 52 weeks of treatment, patients taking setmelanotide lost, on average, 7.9% of their BMI. 61% of patients lost ≥5% of their BMI, and 39% lost ≥10%.

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