Rhythm Pharmaceuticals stock price rises due to FDA approval

Investing.com – Rhythm Pharmaceuticals (NASDAQ: RYTM) stock price rose 11% on Friday morning after the FDA approved IMCIVREE (setmelanotide) for the treatment of acquired hypothalamic obesity.

This 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 for reducing overweight and obesity in adults and children aged 4 and older, and for maintaining weight loss over the long term.

The approval was based on results from the global Phase 3 TRANSCEND trial, which involved 142 patients with acquired hypothalamic obesity. The study met its primary endpoint, showing a significant reduction in body mass index (BMI) of 18.4% compared to placebo. At 52 weeks, participants treated with setmelanotide achieved a 15.8% reduction in BMI, while the placebo group experienced a 2.6% increase.

Acquired hypothalamic obesity is most commonly seen after tumors and their treatment, or following other hypothalamic injury or dysfunction. Rhythm estimates that approximately 10,000 people in the U.S. have acquired hypothalamic obesity.

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

IMCIVREE is also approved in the U.S. and Europe for adults and children aged 2 and older with syndromic or monogenic obesity caused by Bardet-Biedl syndrome or deficiencies in pro-opiomelanocortin, prohormone convertase 1, or leptin receptor.

The company stated that IMCIVREE will be immediately available to patients in the United States.

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