Rhythm's Setmelanotide trial published in NEJM shows 19.8% placebo-adjusted BMI reduction in hypothalamic obesity

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Rhythm Pharmaceuticals announced that results from its pivotal Phase 3 TRANSCEND trial of Setmelanotide in patients with acquired hypothalamic obesity have been published in The New England Journal of Medicine. The study, the largest and longest placebo-controlled trial in this population, showed that over 52 weeks, patients treated with Setmelanotide achieved a placebo-adjusted BMI reduction of 19.8%, with a mean BMI decrease of 16.5% compared to a 3.3% increase in the placebo group. 80% of treated patients achieved at least a 5% BMI reduction, alongside clinically meaningful improvements in hunger. In March 2026, the U.S. FDA approved Setmelanotide, marketed as IMCIVREE, as the first and only therapy for acquired hypothalamic obesity in adults and children aged four years and older, and the European Medicines Agency also recommended marketing authorization for this indication.

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Phase 3 trial results published in NEJM show significant BMI reduction and FDA/EMA approval for hypothalamic obesity.

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