Feel the Rhythm, Rhythm Pharmaceuticals (NASDAQ: RYTM) stock jumps on FDA approval

Rhythm Pharmaceuticals (NASDAQ: RYTM) shares rose 11% Friday morning following FDA approval of IMCIVREE (setmelanotide) for patients with acquired hypothalamic obesity.

The approval marks the first and only FDA-approved therapy for acquired hypothalamic obesity, a rare disease characterized by accelerated and sustained weight gain caused by hypothalamic injury or dysfunction. IMCIVREE is indicated to reduce excess body weight and maintain reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity.

The approval is based on results from the global Phase 3 TRANSCEND trial involving 142 patients with acquired hypothalamic obesity. The study met its primary endpoint, with a statistically significant -18.4% placebo-adjusted reduction in body mass index. Study participants on setmelanotide therapy achieved a -15.8% BMI reduction compared with a +2.6% increase among patients on placebo at 52 weeks.

Acquired hypothalamic obesity most frequently follows tumors and their treatment or other hypothalamic injury or dysfunction. Rhythm estimates there are approximately 10,000 people living with acquired hypothalamic obesity in the U.S.

The most common adverse reactions observed in the trial were skin hyperpigmentation, nausea, vomiting and headache.