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Setmelanotide receives orphan drug designation for treatment of Prader-Willi syndrome
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The FDA granted orphan drug designation to setmelanotide, a melanocortin-4 receptor (MC4R) agonist, for the treatment of Prader-Willi syndrome, according to a press release from Rhythm.
Setmelanotide (RM-493, Rhythm), is a first-in-class MC4R agonist in development for the treatment of obesity caused by genetic deficiencies in the MC4 pathway, a key pathway in humans that regulates energy expenditure, homeostasis, and appetite.
Phase 2 trials for the drug are ongoing to treat both Prader-Willi syndrome and pro-opiomelanocortin deficiency obesity, a rare, life-threatening genetic disorder of the MC4 pathway associated with appetite and obesity.
"The orphan drug designation is an important regulatory milestone as we advance development of setmelanotide for the treatment of Prader-Willi syndrome," Keith Gottesdiener, CEO of Rhythm, said in a press release. "Phase 2 trials are now under way for both PWS and [pro-opiomelanocortin] deficiency obesity, and we expect to complete these trials in the first half of 2016."
The FDA's Orphan Drug Designation program is designed to facilitate drug development for rare diseases, providing substantial benefits to the sponsor, including the potential for grants to fund clinical studies, study-design assistance and several years of market exclusivity for the product upon regulatory approval.