Rhythm Pharmaceuticals Announces FDA Acceptance of New Drug Application for Setmelanotide for the Treatment of POMC and LEPR Deficiency Obesities
– FDA grants Priority Review of application and sets PDUFA goal date of
“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
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.
About POMC and LEPR Deficiency Obesities
POMC and LEPR deficiency obesities are ultra-rare genetic disorders. Rhythm estimates there are approximately 100 to 500 patients in the
Setmelanotide is an investigational, 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 and LEPR deficiency obesity. The
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 our expectations surrounding the PDUFA goal date and the timing for submission of an MAA. 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 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, the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies, clinical trials and commercialization prospects, and general economic conditions, and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.
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Source: Rhythm Pharmaceuticals, Inc.