Rhythm Pharmaceuticals Announces The FDA Has Accepted Its Supplemental New Drug Application For Setmelanotide To Treat Acquired Hypothalamic Obesity

Author: Benzinga Newsdesk | August 20, 2025 03:03pm

U.S. Food and Drug Administration accepts sNDA for filing with priority review; sets PDUFA goal date of December 20, 2025 --

– European Medicines Agency accepts and validates submission for Type II variation MAA for setmelanotide to treat acquired hypothalamic obesity –

– Company to host "Commercial Readiness for Acquired Hypothalamic Obesity," event for investors and analysts on Wednesday, September 24, in Boston –

Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the company's supplemental New Drug Application (sNDA) for setmelanotide seeking approval for the treatment of conditions associated with acquired hypothalamic obesity. The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2025.

Additionally, the European Medicines Agency (EMA) has confirmed validation of the Type II variation submission to the Marketing Authorization Application (MAA) for setmelanotide for the same indication. The application review began on August 16, 2025, by the Committee for Medicinal Products for Human Use (CHMP), which will issue an opinion to the European Commission (EC) regarding potential approval.

Setmelanotide is a melanocortin-4 receptor (MC4R) agonist, previously approved as IMCIVREE® by the FDA, the EMA, and UK's Medicines & Healthcare Products Regulatory Agency (MHRA) for obesity due to Bardet-Biedl syndrome and POMC, PCSK1 or LEPR deficiencies.

"Taken together, these important milestones represent a crucial step in our urgent mission to bring forward the first approved treatment for patients with acquired hypothalamic obesity," said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. "Treatment approaches indicated for general obesity offer limited long-term efficacy for patients living with acquired HO, who face unique challenges including reduced energy expenditure and increased hunger or hyperphagia specifically resulting from MC4R pathway impairment due to hypothalamic injury. We're committed to working closely with regulators to make setmelanotide available to the patient community as quickly as possible."

The sNDA and Type II Variation are supported by statistically significant and clinically meaningful data from the pivotal Phase 3 TRANSCEND trial of setmelanotide in 120 patients with acquired hypothalamic obesity. Topline results from TRANSCEND, the largest and longest randomized, placebo-controlled trial in acquired hypothalamic obesity to date, were presented in an oral session at the Endocrine Society's Annual Meeting in April 2025. The global study met its primary endpoint, with a statistically significant -19.8% placebo-adjusted reduction in body mass index (BMI). For the primary endpoint of mean BMI change from baseline, study participants on setmelanotide therapy (n=81) achieved a -16.5% reduction compared with a +3.3% increase among patients on placebo (n=39) at 52 weeks (p<0.0001). Adult patients (age 18 and older; n=49) achieved a -19.2% placebo-adjusted BMI reduction at 52 weeks, and pediatric patients (younger than age 18; n=71) achieved a -20.2% placebo-adjusted BMI reduction at 52 weeks. Setmelanotide was generally well tolerated in the TRANSCEND study. The most common treatment-emergent adverse events (affecting >20% of participants) were nausea, vomiting, diarrhea, injection site reaction, skin hyperpigmentation and headache.

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