WEDNESDAY, July 22, 2026 (HealthDay News) -- For patients aged 4 to 66 years with acquired hypothalamic obesity, setmelanotide leads to significantly greater reductions in body mass index (BMI) and hunger than placebo, according to a study published in the July 9 issue of the New England Journal of Medicine.
Jennifer L. Miller, M.D., from the University of Florida in Gainesville, and colleagues conducted a phase 3 trial involving participants at least 4 years of age with acquired hypothalamic obesity who were randomly assigned to receive setmelanotide (at a dose of 1.5 to 3.0 mg) or placebo administered subcutaneously once daily for 52 weeks after a dose-escalation period (81 and 39 participants, respectively). The mean age was 19.9 ± 13.8 years (range, 4 to 66).
The researchers found that the least-squares mean (LSM) change in BMI at 52 weeks was −16.5 and 3.3 percent with setmelanotide and placebo, respectively, and the LSM change in the weekly average of maximal daily hunger scores was −2.73 and −1.45, respectively. Overall, 100 and 90 percent of participants in the setmelanotide and placebo groups reported adverse events; serious adverse events were reported by 28 and 8 percent, respectively. Skin hyperpigmentation, nausea, vomiting, and headache were the most common adverse events with setmelanotide.
"This approval represents years of collaboration among researchers, physicians, patients, and families around the world," coauthor Reema Habiby, M.D., from Lurie Children's Hospital in Chicago, said in a statement. "We're excited to be able to offer a treatment that addresses the biology of this disease rather than simply trying to manage its symptoms."
Several authors disclosed ties to biopharmaceutical companies, including Rhythm Pharmaceuticals, the manufacturer of setmelanotide; one author served as an expert witness for a law firm.
