Rhythm Pharmaceuticals, Inc. (RYTM) 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 (NEJM).
Acquired hypothalamic obesity is a rare condition that can occur following hypothalamic injury, leading to accelerated and sustained weight gain. Patients often face limited treatment options, making effective therapies a critical need.
The TRANSCEND study, the largest and longest placebo-controlled trial conducted in this patient population, enrolled adults and children aged four years and older. 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. Notably, 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 (IMCIVREE) as the first and only therapy for acquired hypothalamic obesity in adults and children aged four years and older. The European Medicines Agency also recommended marketing authorization for this indication.
Dr. Christian Roth, senior author and pediatric endocrinologist at Seattle Children’s Research Institute, emphasized the importance of these findings, noting the consistent reductions in BMI and hunger as potentially transformative for patients and families living with hypothalamic obesity.
