Rhythm Pharmaceuticals Completes Rolling Submission of New Drug Application to U.S. Food and Drug Administration for Setmelanotide in POMC and LEPR Deficiency Obesities
“The completion of our first NDA submission marks an integral first step in our journey to transform the care of people living with not only POMC and LEPR deficiency obesities, but also many other rare genetic disorders of obesity,” said
The FDA typically has a 60-day filing review period to determine whether the NDA is sufficiently complete and acceptable for filing. Rhythm has requested priority review for the application which, if granted, could provide a target FDA review period of six-months from the application filing date.
As first reported in
About POMC and LEPR Deficiency Obesities
POMC and LEPR deficiency obesities are ultra-rare disorders. Rhythm estimates there are approximately 100 to 500 patients in the
Setmelanotide is an investigational, potent melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates energy expenditure and appetite. Variants in genes may impair the function of the MC4R pathway, potentially leading to insatiable hunger and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to restore the function of an impaired MC4R pathway and, in so doing, reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions. The FDA has 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, LEPR deficiency obesity, Bardet-Biedl syndrome (BBS) and Alström syndrome. The European Medicines Agency 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. Both the FDA and EMA have granted orphan drug status to setmelanotide for POMC and LEPR deficiency obesities, BBS and Alström syndrome.
Rhythm is a late-stage biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. In
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of setmelanotide, including the effectiveness of setmelanotide in patients with POMC and LEPR deficiency obesities, and the timing for approval of an NDA. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the impact of the COVID-19 outbreak and other global economic factors, the impact of our management transition, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, our liquidity and expenses, and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended
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Source: Rhythm Pharmaceuticals, Inc.