Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Study of Setmelanotide in Patients Between 2 and 5 Years Old in The Lancet Diabetes & Endocrinology

Core Insights - Rhythm Pharmaceuticals announced positive results from its Phase 3 VENTURE trial for setmelanotide, targeting severe obesity in children under 5 with specific genetic deficiencies [1][2] - The trial showed significant reductions in body mass index (BMI) and hunger levels among participants, indicating the potential effectiveness of setmelanotide in this demographic [2][3] Company Overview - Rhythm Pharmaceuticals is a biopharmaceutical company focused on rare neuroendocrine diseases, with its lead product, IMCIVREE (setmelanotide), approved for chronic weight management in patients with specific genetic obesity conditions [5][6] - Setmelanotide has received regulatory approvals in both the U.S. and the European Union for treating obesity and controlling hunger in genetically confirmed cases of Bardet-Biedl syndrome (BBS) and other deficiencies [4][7] Clinical Trial Results - The Phase 3 VENTURE trial enrolled 12 patients, with 83% achieving a ≥0.2-point reduction in BMI Z-score and an 18% mean percent change in BMI from baseline [2][3] - Caregivers reported a 91% reduction in hunger among patients, alongside a decrease in personal burden [2] Safety and Tolerability - Setmelanotide was generally well tolerated, with no serious adverse events leading to discontinuation or death reported [3] - Common treatment-emergent adverse events included skin hyperpigmentation (75%), vomiting (58%), and nasopharyngitis (42%) [3][16] Regulatory Developments - Rhythm has submitted a supplemental New Drug Application (sNDA) to the FDA to expand the label of IMCIVREE for pediatric patients aged 2 to under 6 years [4] - The FDA has granted Priority Review for the sNDA, with a Prescription Drug User Fee Act (PDUFA) goal date set for December 26, 2024 [4]