Setmelanotide in Bardet‐Biedl Syndrome: A 52‐Week Comparison of Phase 3 Trial Participants With a Matched Registry Cohort
An erratum to this article was published (Obesity (Silver Spring), Jun 2026 ↗)
Authors' conclusion
This indirect comparison provides additional strong evidence that setmelanotide significantly improves weight outcomes in patients with BBS. These findings further support its clinical benefit over 52 weeks in managing obesity associated with BBS.
Study context
- Population
- Patients with Bardet-Biedl syndrome (BBS) ≥ 6 years of age
- Participants
- 29 vs 58
- Exposure
- Setmelanotide vs matched CRIBBS registry controls
- Setmelanotiden = 29
- Controln = 58reference
- Outcome
- Responder rate
- Follow-up
- Week 52
Methodological detailspropensity-score matched, external natural history control, indirect comparison
- Propensity-score matched
- External natural history control
- Indirect comparison
Main finding
As reported in the abstract
P < 0.001
Observational data: associations, not effects.
Secondary outcomes
Responder rate, pediatric patients · week 52
P < 0.001
Responder rate, adults · week 52
P = 0.001
Changes in BMI, BMI z-score and body weight
- Secondary outcomes demonstrated consistent benefits of setmelanotide treatment
Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.
Original publication
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