Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for IMCIVREE (setmelanotide) to include children as young as 2 years old. IMCIVREE is indicated to reduce excess body weight and maintain weight reduction long-term in patients 2 years of age 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 and POMC, PCSK1 and LEPR deficiencies are rare melanocortin-4 receptor (MC4R) pathway diseases with hallmark characteristics that include hyperphagia, or pathological, insatiable hunger and impaired satiety accompanied by persistent and abnormal food-seeking behaviors, and early-onset obesity. IMCIVREE is the first and only precision medicine to target impairment of the hypothalamic MC4R pathway, a root cause of hyperphagia and obesity due to BBS and POMC, PCSK1 and LEPR deficiencies in adults and children as young as 2 years old in the United States, as well as Europe.