Rhythm announces FDA approval of Imcivree for patients as young as 2

Rhythm Pharmaceuticals (RYTM) announced that the U.S. Food and Drug Administration has approved an expanded indication for IMCIVREE 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 or genetically confirmed pro-opiomelanocortin, including proprotein convertase subtilisin/kexin type 1, deficiency or leptin receptor deficiency. BBS and POMC, PCSK1 and LEPR deficiencies are rare melanocortin-4 receptor 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. “It’s important to understand that rare MC4R pathway diseases differ from general obesity as the insatiable hunger these patients experience is pathologic and a result of impairment to a pathway in the brain. With this insatiable hunger, most patients develop early-onset obesity before the age of 5,” said Ilene Fennoy, MD, MPH, pediatric endocrinologist, obesity specialist and professor of Pediatrics at Columbia University Medical Center. “Obesity in childhood, if untreated, can lead to a greater risk of severe and long-term health complications, making early intervention to treat obesity critical. With this expanded indication for IMCIVREE, patients now can receive a much needed, targeted treatment that we believe can address a root cause of their obesity at a very young age.”