ODP606 Long-term Efficacy of Setmelanotide in Patients With Bardet-Biedl Syndrome

ABSTRACT: Abstract

Objective

Bardet-Biedl syndrome (BBS) is a rare genetic disease characterized by hyperphagia (pathologic insatiable hunger) and early-onset, severe obesity believed to be driven by impaired signaling in the melanocortin-4 receptor (MC4R) pathway. In a Phase 2 and a pivotal Phase 3 trial, treatment with the MC4R agonist setmelanotide produced beneficial reductions in weight, body mass index (BMI), BMI Z score, and hunger in patients with BBS at ∼1 year. The current analysis is the first to assess the continued long-term efficacy of setmelanotide administration in patients with BBS over ∼2 years.

Methods

Patients with BBS aged ≥6 years were eligible for this observational long-term extension (LTE) trial (NCT03651765) if they completed an index trial in which they r

SUBMITTER:  

PROVIDER: S-EPMC9624643 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature