FDA Breakthrough status for Rhythm's setmelanotide

The Food and Drug Administration has expanded a previously granted Breakthrough Therapy designation (BTD) for setmelanotide, a novel melanocortin-4 receptor (MC4R) agonist from Boston, USA-based biotech firm Rhythm Pharmaceuticals.

The expanded BTD is for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway (the MC4 pathway), which includes both pro-opiomelanocortin (POMC) and leptin receptor (LepR) deficiency obesity. The FDA had previously granted BTD to setmelanotide for the treatment of POMC deficiency obesity.