SAT-703 - Real-Word Weight and Healthcare Utilization Outcomes with Setmelanotide in US Patients With Bardet-Biedl Syndrome

Background: Early-onset obesity substantially contributes to the clinical and economic burden for patients with Bardet-Biedl Syndrome (BBS). This study evaluated the real-world effectiveness of setmelanotide on weight-related outcomes and healthcare resource utilization among US patients with obesity due to BBS. A secondary analysis assessed the impact of delayed setmelanotide initiation.

Methods: This retrospective single-arm cohort study used de-identified specialty pharmacy data linked at the tokenized patient level with Komodo’s Healthcare Map© claims data (2022-2025). Eligible patients were required to be ≥6 years of age, have no evidence of prior setmelanotide use, have ≥ 6 months’ enrollment prior to initiating setmelanotide, and ≥12 prescription fills in a 12-month follow-up period (compliant use). For the secondary analysis, all inclusion criteria applied but patients were also required to be ≥6 years of age on June 16, 2022 (setmelanotide approval date).

Results: A total of 286 patients met the inclusion criteria for the primary analysis. Mean (SD) age was 27.83 (16.45) years, 66% were male, and mean body mass index (BMI) among adults was 45.75 (12.49) kg/m2 in adults (≥18 years). After 12 months of setmelanotide treatment, 62% of adults achieved ≥10% body weight loss; 52.1% of all patients (pediatric and adult) achieved this threshold. Mean percent body weight loss was -9.8% in adults and -7.8% across all patients, with a statistically significant reduction in BMI observed among adult patients (p < 0.05). There was a significant reduction in obesity-related outpatient healthcare utilization rates following treatment initiation (rate difference: -1.03; p< 0.05) with additional reduction in all-cause outpatient utilization.

In the secondary analysis (n= 653), patients were stratified by quartiles of time to setmelanotide initiation. Compared with those initiating treatment earliest, patients who took the longest to initiate setmelanotide weighed 20.75% more (128.01 vs. 106.27 kg), had a 13.36% higher BMI (49.2 vs. 43.4 kg/m2), and demonstrated higher rates of obesity-related outpatient healthcare utilization.

Conclusions: Among US patients with obesity due to BBS, setmelanotide treatment was associated with significant improvements in weight outcomes and decreased healthcare utilization in real-world clinical settings. These findings demonstrate the effectiveness of sustained setmelanotide treatment in a population with severe, progressive obesity and high healthcare resource use.

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