Rhythm Pharmaceuticals, Inc. announced that patients with Bardet-Biedl syndrome (BBS) and obesity who participated in the company's global Phase 3 clinical trial of setmelanotide reported clinically meaningful improvements across multiple health-related quality of life (HRQOL) measures based on an analysis published in the Orphanet Journal of Rare Diseases. In total, 20 patients with BBS and obesity completed various self-reported HRQOL measures at baseline and after 52 weeks of treatment with setmelanotide, which is a precision medicine designed to re-establish MC4R pathway signaling that is approved by the U.S. Food & Drug Administration (FDA) for chronic weight management in adult and pediatric patients six years of age and older with monogenic or syndromic obesity due to BBS. For the 52-week analysis, 9 patients younger than 18 years, or their caregiver, reported a significant and clinically meaningful change in their quality of life with a mean change from baseline of +11.2 points on the Pediatric Quality of Life Inventory (PedsQL) measure.

In addition, 11 adult patients who completed the Impact of Weight on Quality of Life Questionnaire-Lite (IWQOL-Lite) achieved a significant and clinically meaningful improvement in their weight related quality of life with a mean change from baseline of +12.0 points. IWQOL-Lite score was significantly correlated with changes in percent body weight (P=0.0037) and body mass index (P=0.0098).