Targeted Therapy Reverses Weight Gain From Rare Cause of Obesity

In a randomized trial, the MC4R agonist setmelanotide reduced BMI and hunger in patients with acquired hypothalamic obesity.After 1 year, participants lost 36.4 lb with setmelanotide but gained 5.3 lb on placebo.The findings supported setmelanotide's recent label expansion.

Treatment with setmelanotide (Imcivree), a melanocortin-4 receptor (MC4R) agonist, led to significant reductions in body mass index (BMI) and hunger in individuals with acquired hypothalamic obesity, the phase III TRANSCEND trial showed.

By week 52, adult and pediatric participants as young as 4 years who received setmelanotide had a mean BMI reduction of 16.5% compared with a 3.3% increase in the placebo group (P<0.001), reported Christian Roth, MD, of Seattle Children's Research Institute in Washington, and colleagues.

This equated to 36.4 lb of weight loss with setmelanotide compared with a 5.3 lb increase with placebo. Treatment also reduced the weekly average of maximum daily hunger scores (-2.73 vs -1.45 points, P=0.009), the authors wrote in the New England Journal of Medicine.

Based on the trial data, the once-daily injection was approved earlier this year as the first therapy specifically for acquired hypothalamic obesity in patients 4 years and older.