Rhythm Pharmaceuticals has received an expanded indication for IMCIVREE (setmelanotide) to include children as young as 2 years old 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 behaviours, and early-onset obesity. The company claims 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 Dr Ilene Fennoy, 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.”
Results from clinical trials demonstrate that setmelanotide delivers significant and sustained reductions in measures of weight and hunger. Results from the Phase 3 VENTURE trial were published in the peer-reviewed journal The Lancet Diabetes & Endocrinology in November 2024. The most common adverse events are skin hyperpigmentation, injection site reactions, diarrhoea, nausea and headache.
“Today’s approval is welcome news for the BBS community and others with rare MC4R diseases who struggle with hyperphagia,” said Tim Ogden, President of the Bardet Biedl Syndrome Foundation. “Many children with BBS feel hungry or think about food regardless of how much or how recently they’ve eaten, leaving families to deal with children sneaking or stealing food, which makes daily life extremely stressful. Parents have enough to worry about and manage when their child has a multi-systemic syndrome like BBS and IMCIVREE can be an important tool for their obesity.”
IMCIVREE initially received approval from the FDA in November 2020 for patients 6 years old and older with POMC, PCSK1 or LEPR deficiencies and approval in June 2022 for use in patients with BBS. IMCIVREE has also received marketing authorization from the United Kingdom’s Medicines & Healthcare products Regulatory Agency (MHRA) and the European Commission (EC) for use in patients as young as 2 years of age.
Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective:
Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign
Other types of obesity not related to BBS or POMC, PCSK1, or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity
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