Rhythm Pharmaceuticals Announces FDA Review of sNDA for Setmelanotide in Acquired Hypothalamic Obesity
Thursday, August 21, 2025
Rhythm Pharmaceuticals, Inc. a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, has reported that the US Food and Drug Administration (FDA) has accepted for review its supplemental New Drug Application (sNDA) for setmelanotide in acquired hypothalamic obesity. The submission has been granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) goal date set for 20 December 2025.
In parallel, the European Medicines Agency (EMA) has validated a Type II variation to the Marketing Authorisation Application (MAA) for setmelanotide for the same indication. The Committee for Medicinal Products for Human Use (CHMP) began its review on 16 August 2025 and will provide an opinion to the European Commission (EC) on possible approval.
Setmelanotide, a melanocortin-4 receptor (MC4R) agonist, is already approved as IMCIVREE® by the FDA, EMA and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for obesity linked to Bardet-Biedl syndrome and POMC, PCSK1 or LEPR deficiencies.
The new regulatory submissions are supported by results from the Phase 3 TRANSCEND trial, which involved 120 patients with acquired hypothalamic obesity. This global, placebo-controlled study is the largest and longest of its kind in this condition. The trial achieved its primary endpoint, showing a statistically significant placebo-adjusted reduction in body mass index (BMI) of –19.8%.
Patients receiving setmelanotide therapy (n=81) recorded a mean BMI reduction of –16.5% over 52 weeks, compared with a +3.3% increase in the placebo group (n=39; p<0.0001). Adult participants (n=49) achieved a –19.2% placebo-adjusted reduction, while paediatric participants (n=71) achieved a –20.2% reduction at 52 weeks.
Setmelanotide was generally well tolerated. The most common treatment-related side effects, seen in more than 20% of patients, included nausea, vomiting, diarrhoea, injection site reactions, skin hyperpigmentation and headache.
Source: globenewswire.com
