Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trials of Setmelanotide in The Lancet Diabetes & Endocrinology - Seite 2

“These results are significant because, as we know from natural history data, individuals living with POMC or LEPR deficiency obesity consistently experience substantial weight gain each year beginning in early childhood, and we would not expect any of these patients to be able to achieve 10 percent weight loss over the course of a year without continued treatment,” said co-author Karine Clément, professor of nutrition at Pitié-Salpêtrière hospital and Sorbonne University in Paris. “These data and the significant unmet need to address the obesity and hyperphagia caused by rare genetic disorders of obesity underscore the importance of testing for genetic variants that may impair MC4R activation and lead to severe obesity.”

In May 2020, Rhythm announced that the U.S. Food and Drug Administration (FDA) accepted the company's New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity, granted Priority Review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of November 27, 2020. In July 2020, the Company announced the submission of its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the treatment of POMC deficiency obesity and LEPR deficiency obesity.

“We are grateful to the authors and the investigators involved in our pivotal Phase 3 clinical trials for their continued partnership in advancing setmelanotide to address significant unmet needs facing people with rare genetic disorders of obesity,” Murray Stewart, M.D., Chief Medical Officer of Rhythm, said.

The article is available online here: http://www.thelancet.com/journals/landia/article/PIIS2213-8587(20)3036 ....To request a copy of the article, email [e-mail].

About Setmelanotide
Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions.