The US FDA has approved Setmelanotide, a melanocortin-4 receptor (MC4R) agonist, is the first FDA-approved therapy for BBS, a rare genetic disorder that impairs a hunger signal along the melanocortin-4 receptor (MC4R) pathway.
Individuals with Bardet-Biedl Syndrome typically have obesity that starts at age 1 along with insatiable hunger (hyperphagia). Available weight management options are generally unsuccessful. Other symptoms may include retinal degeneration, reduced kidney function, or extra digits of the hands or feet.
The most common adverse reactions (with an incidence ≥ 20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection.