FDA Grants Priority Review for Rhythm Pharmaceuticals’ Setmelanotide in Hypothalamic Obesity

FDA Approved Rhythm’s sNDA

Announcement

Rhythm Pharmaceuticals Inc., a leading commercial-stage biopharmaceutical company aiming to change the lives of patients with rare neuroendocrine disease, has been given permission to file the supplemental new drug application (sNDA) for setmelanotide by the US Food and Drug Administration. The filing will lead to approval of the treatment of hypothalamic obesity conditions. The FDA has initiated a priority review of the sNDA and hired a Prescription Drug User Fee Act (PDUFA), expecting to clear by 20th December 2025.

Also, the European Medicines Agency (EMA) has green signaled validation of the type 2 variation submission to the marketing authorization application (MAA) for setmelanotide for the same indication. On 16th August 2025, the application review will begin with the Committee for Medicinal Products for Human Use (CHMP). This will create an opinion for the European Commission (EC) of the approval.

sNDA Potential

The type 2 variation and sNDA are contributed by calculated prominent and clinically oriented data from the pivotal phase 3 TRANSCEND trial of setmelanotide in 120 patients with attained hypothalamic obesity. The popular results from TRANSCEND, the largest randomized, placebo-controlled trial in obtained hypothalamic obesity to date, were showcased during an oral session at the Endocrine Society’s annual meeting in April 2025. The global study successfully met its primary endpoint, with a statistically significant 19.8% placebo-adjusted decrease in body mass index (BMI). The other calculation considers the age factor and the duration of the study showed tolerability. The adverse events of the most common treatments affecting participants were vomiting, nausea, skin hyperpigmentation, headache, injection site reaction, and diarrhea.

Further, the acquired hypothalamic obesity is ready for commercialization, with Rhythm hosting the ‘commercial readiness for acquired hypothalamic obesity’ event for analysts and investors on 24th September 2025 from 8:30 am to 11:30 am ET in Boston, MA, to review the US launch intentions.

Views and Statements from the Company Leaders

MD chairman, chief executive officer, and president of Rhythm, David Meeker, said, “This special milestone is a vital step in our emergency mission to introduce the first-ever approved treatment for hypothalamic obesity patients. The treatment approaches for general obesity enable limited long-term efficacy for patients with HO, who encounter different challenges like increased hunger or hyperphagia and decreased energy expenditure. This is generated from the MC4R pathway impairment due to hypothalamic injury. We’re dedicated to working with regulators to make setmelanotide accessible to the patient population quickly.”