The U.S. Food and Drug Administration (FDA) on Thursday approved an expanded indication for Rhythm Pharmaceuticals Inc.’s Imcivree (setmelanotide) to treat acquired hypothalamic obesity (HO).

The FDA also approved Imcivree (setmelanotide) in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome or Pro-opiomelanocortin, proprotein convertase subtilisin/kexin type 1, or leptin receptor deficiency.

FDA Expands Imcivree Use To Rare Hypothalamic Obesity

Acquired HO is a rare disease characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus or hypothalamic dysfunction.

With this label expansion, Imcivree is indicated to reduce excess body weight and maintain the reduction long-term in adults and pediatric patients aged 4 years and older with acquired HO.

“Imcivree is now the first and only FDA-approved therapy for acquired HO…,” said David Meeker, Chairman, CEO, and President of Rhythm.

The MC4R pathway is responsible for controlling physiological functions such as energy expenditure, hunger, and weight regulation. Acquired HO most frequently follows tumors and their treatment or other hypothalamic injury or dysfunction.

Based on analysis of the literature, tumor registries, and claims data, Rhythm estimates there are approximately 10,000 people living with acquired HO in the U.S.

Phase 3 TRANSCEND Trial Supports Approval

The approval is supported by the positive pivotal Phase 3 TRANSCEND trial of setmelanotide in 142 patients.

The global study met its primary endpoint, with a statistically significant 18.4% placebo-adjusted reduction in body mass index (BMI).

For the primary endpoint of mean BMI change from baseline, study participants on setmelanotide therapy (n=94) achieved a -15.8% reduction compared with a +2.6% increase among patients on placebo (n=48) at 52 weeks.

EMANATE Trial Misses Endpoint, Shows Subgroup Benefit

On Tuesday, Rhythm Pharmaceuticals ( RYTM ) said the EMANATE trial of setmelanotide in patients with rare, genetically driven obesities of the MC4R pathway did not meet the primary endpoints.

The post hoc analyses indicated that setmelanotide achieved statistically significant BMI reductions in patients with obesity due to specific genetic variants at the 52-week mark.

RYTM Price Action: Rhythm Pharmaceuticals ( RYTM ) shares were up 8.34% at $97.84 during premarket trading on Friday, according to Benzinga Pro data.

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