David Meeker, MD, Chief Executive Officer of Rhythm Pharmaceuticals, discusses the U.S. Food and Drug Administration’s (FDA) approval of setmelamotide for children 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency.
BBS, POMC, PCSK1, and LEPR deficiencies are rare melanocortin-1 receptor (MC4R) pathway diseases. Common characteristics include hyperphagia, impaired satiety, persistent and abnormal food-seeking behaviors, and early-onset obesity.
Setmelanotide, or Imcivree, is an MC4R agonist that targets the impairment in the hypothalamic MC4R pathway. Activation of this pathway helps increase satiety signals and energy expenditure.
The approval follows results of clinical trials where setmelanotide was observed to significantly and sustainably reduce measures of weight and hunger. Safety and tolerability has also been favorable, with common adverse events being skin hyperpigmentation, injection site reactions, diarrhea, nausea, and headache.
The treatment was originally approved in November 2020 for patients 6 years and older with POMC, PCSK1, and LEPR deficiencies, and June 2022 for patients with BBS.
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To learn more about these deficiencies and other rare metabolic disorders, visit https://checkrare.com/diseases/metabolic-disorders/
