Background Setmelanotide, a melanocortin-4 receptor (MC4R) agonist, has been shown to reduce hunger and weight
in patients aged 6 years and older with proopiomelanocortin (POMC) deficiency (including biallelic variants in
proprotein convertase subtilisin/kexin type 1 [PCSK1]), leptin receptor (LEPR) deficiency, or Bardet-Biedl syndrome
(BBS). No approved therapies for patients younger than 6 years old currently exist. The phase 3, open-label VENTURE
trial aimed to evaluate the efficacy and safety of setmelanotide in patients aged 2–5 years with POMC or LEPR
deficiency or BBS
Setmelanotide in patients aged 2–5 years
