Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients as Young as 2 Years Old

Core Viewpoint - Rhythm Pharmaceuticals has received FDA approval for an expanded indication of IMCIVREE (setmelanotide) to treat obesity in children as young as 2 years old, specifically for those with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), PCSK1, or LEPR deficiencies [5][22][29] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, headquartered in Boston, MA [3][7] - The company is committed to advancing care and precision medicines that address the root causes of these diseases [7] Product Information - IMCIVREE is the first and only precision medicine targeting the hypothalamic MC4R pathway, addressing hyperphagia and obesity in patients with BBS and POMC, PCSK1, or LEPR deficiencies [14] - The drug is indicated for long-term weight reduction in patients aged 2 years and older [17][22] Clinical Development - Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases and investigational MC4R agonists [3] - Results from the Phase 3 VENTURE trial indicate significant and sustained reductions in weight and hunger [31] Market Impact - The approval is seen as a significant advancement for the BBS community, providing a targeted treatment option for children suffering from insatiable hunger and obesity [15] - Approximately 4,000 to 5,000 people in the U.S. are affected by BBS, while POMC, PCSK1, and LEPR deficiencies affect around 600 to 2,500 individuals [16]