We are studying rare genetic disorders of obesity.
An investigational, melanocortin-4 receptor (MC4R) agonist in clinical development for the treatment of rare genetic disorders of obesity
For more information about Rhythm’s clinical trials, please visit clinicaltrials.gov
Rhythm’s lead product candidate, setmelanotide, is an MC4R agonist designed to restore impaired MC4R pathway function caused by genetic variants that occur upstream of the MC4R. Setmelanotide has the potential to affect weight loss and reduce hunger, and it is specifically in clinical development for individuals affected by rare genetic disorders of obesity.
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic variants upstream of the MC4R in the MC4R pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl syndrome (BBS), and Alström syndrome. Also, the FDA has granted Orphan Drug Designation for setmelanotide for the treatment of POMC deficiency obesity, LEPR deficiency obesity and Bardet-Biedl syndrome. The European Medicines Agency (EMA) has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. The EMA’s Committee for Orphan Medicinal Products issued a positive opinion recommending setmelanotide for designation as an orphan medicinal product for the treatment of patients with BBS.
Proof-of-concept has been established with setmelanotide in POMC deficiency obesity, LEPR deficiency obesity, BBS, and Alström syndrome, and Rhythm is currently evaluating setmelanotide in pivotal trials for these indications:
- Rhythm’s pivotal, phase 3 studies of setmelanotide in POMC and LEPR deficiency obesity met their primary and key secondary endpoints with statistically significant and clinically meaningful results, and the company expects to submit its New Drug Application to the FDA in the fourth quarter of 2019 or first quarter of 2020
- The Company expects to submit its Marketing Authorization Application to EMA following completion of the submission to the FDA.