Setmelanotide: a promising advancement for pediatric patients with rare forms of genetic obesity.

ABSTRACT:

Purpose of review

Examine Setmelanotide use in patients with rare genetic variants that disrupt the melanocortin pathway.

Recent findings

Between February 2017 and September 2018, 10 participants with pro-opiomelanocortin (POMC)/ proprotein convertase subtilisin/kexin type 1 (PCSK1) deficiency and 11 participants with leptin receptor (LEPR) deficiency were enrolled in open-label, phase 3 trials at 10 centers in the United States and internationally to assess the efficacy and safety of the melanocortin-4 receptor (MC4R) agonist Setmelanotide. 80% of POMC participants and 45% of LEPR participants achieved at least 10% weight loss at 1 year. Significant changes in hunger scores were seen for both cohorts as well. Setmelanotide was well tolerated with injection site reactions an

SUBMITTER: Trapp CM 

PROVIDER: S-EPMC9973437 | biostudies-literature | 2023 Apr

REPOSITORIES: biostudies-literature