The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for setmelanotide for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.
Setmelanotide is a melanocortin-4 receptor (MC4R) agonist that activates the MC4R to regulate energy expenditure and appetite. The MC4R pathway consists of variants in genes associated with early-onset obesity, including POMC and LEPR genes.
The application is supported by data from 2 pivotal multicenter, open-label, single-arm phase 3 studies that assessed the efficacy and safety of setmelanotide in patients aged ≥6 years with either POMC or LEPR deficiency obesity for approximately 1 year. Results from both studies showed that setmelanotide met the primary and all key secondary end points achieving a statistically significant reduction in weight loss and reduction in hunger after 1 year. Overall, mean weight loss was 70.2lbs for patients with POMC deficiency and 36.8lbs for patients with LEPR deficiency.
With regard to safety, treatment-emergent adverse events included injection site reactions, nausea, vomiting, and increased hyperpigmentation.
A Prescription Drug User Fee Act (PDUFA) target date of November 27, 2020 has been set for this application.
“The FDA’s acceptance of our NDA for Priority Review signifies a critical milestone toward our mission of addressing the insatiable hunger and early-onset, severe obesity that affects individuals living with rare genetic disorders of obesity,” said Murray Stewart, MD, Chief Medical Officer of Rhythm Pharmaceuticals. “We are grateful to the Agency for its agility and dedication during these challenging times, as that has enabled continued progress and productive dialogue despite the ongoing pandemic.”
The FDA previously granted Breakthrough Therapy designation to setmelanotide for this indication. The investigational therapy has also received an Orphan Drug designation for the treatment of Alström Syndrome.
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