Shares of Rhythm Pharmaceuticals (NASDAQ:RYTM) climbed 11% on Friday morning after the U.S. Food and Drug Administration granted approval for IMCIVREE (setmelanotide) to treat acquired hypothalamic obesity. The decision designates IMCIVREE as the first and only FDA-approved therapy for this form of obesity, which is driven by hypothalamic injury or dysfunction and marked by rapid, sustained weight gain.
IMCIVREE is indicated to reduce excess body weight and to help maintain that reduction over the long term in both adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity. Rhythm said the treatment will be available to patients in the United States immediately.
The approval was supported by data from the global Phase 3 TRANSCEND trial, which enrolled 142 patients diagnosed with acquired hypothalamic obesity. The trial met its primary endpoint, producing a statistically significant placebo-adjusted reduction in body mass index of -18.4%. At 52 weeks, participants receiving setmelanotide experienced an average -15.8% reduction in BMI, while those on placebo showed a +2.6% increase.
Acquired hypothalamic obesity most commonly follows tumors, their treatment, or other forms of hypothalamic injury or dysfunction. Rhythm estimates there are about 10,000 people in the United States living with the condition.
The TRANSCEND trial also identified safety findings. The most frequently reported adverse reactions among treated patients included:
- skin hyperpigmentation
- nausea
- vomiting
- headache
IMCIVREE already has approved uses in adult and pediatric patients aged 2 years and older for certain syndromic or monogenic forms of obesity. Those indications cover obesity due to Bardet-Biedl syndrome or deficiencies in Pro-opiomelanocortin, proprotein convertase subtilisin/kexin type 1, or the leptin receptor.
From a market perspective, the FDA approval represents an expansion of IMCIVREE's labeled population to include acquired hypothalamic obesity and signals immediate commercial availability in the U.S. While the patient population is estimated to be small, the designation as the first FDA-approved therapy for this condition is a regulatory milestone for the company.
Note: This report presents the trial results, labeled indications, safety profile and the company-provided estimate of patient population as stated by Rhythm Pharmaceuticals. The article does not add facts beyond those reported by the company and the TRANSCEND study outcomes noted above.