Promising Trial Results for Rhythm’s Genetic Obesity Treatment, FDA Decision Awaited

Rhythm Pharmaceuticals shared positive results from its Phase 3 VENTURE trial of setmelanotide, targeting early-onset obesity in young children with Bardet-Biedl syndrome (BBS) or specific genetic deficiencies (POMC, PCSK1, LEPR). Over the 52-week study, patients showed a significant reduction in BMI and decreased hunger, with caregivers also noting a reduction in personal burden. The therapy was generally well tolerated, with common side effects being mild and manageable, such as skin hyperpigmentation and mild respiratory symptoms.

Following promising trial results and recent European approval, Rhythm is seeking to expand setmelanotide’s approval in the U.S. to include younger pediatric patients, with an FDA decision expected by December 2024.