Rhythm Announces NDA Acceptance for Setmelanotide for POMC, LEPR Deficiency Obesities

Rhythm Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has accepted the company's New Drug Application (NDA) for setmelanotide, an investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity. The FDA granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. At this time, the FDA has indicated that it is not planning an advisory committee meeting as part of the NDA review.

“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, M.D., 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. We look forward to working closely together throughout the review process to bring setmelanotide, if approved, to patients.”

A Priority Review designation is granted to drug candidates that may offer significant improvements in the safety or effectiveness of the treatment, prevention or diagnosis of a serious disease. Under Priority Review, the FDA aims to take action on an application within six months, compared to 10 months under standard review. The FDA previously granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4R in the central melanocortin pathway, which includes POMC deficiency obesity and LEPR deficiency obesity.

POMC and LEPR deficiency obesities are ultra-rare genetic disorders. Rhythm estimates there are approximately 100 to 500 patients in the U.S. with POMC deficiency obesity and approximately 500 to 2,000 patients in the U.S. with LEPR deficiency obesity. POMC deficiency obesity is a disorder caused by variants in the POMC or PCSK1 genes that can often lead to severe obesity beginning early in life and insatiable hunger, in addition to endocrine abnormalities, and sometimes red hair and light skin pigmentation. LEPR deficiency obesity is a disorder caused by variants in the LEPR gene that can often lead to severe obesity beginning early in life and insatiable hunger, in addition to endocrine abnormalities. Most patients with POMC or LEPR deficiency obesity experience exponential weight gain in the first months of life, which continues rapidly over the course of their lives. This weight gain cannot be mitigated by diet, exercise or other lifestyle changes, or by existing therapeutic interventions.