Setmelanotide, the world's first breakthrough peptide drug targeting acquired hypothalamic obesity
Publish Time:
2025-08-27
Recently, good news came from Rhythm Pharmaceuticals (NASDAQ: RYTM), headquartered in Boston, USA. This commercial biopharmaceutical company, focused on improving the lives of patients with rare neuroendocrine diseases, announced that its submitted supplemental New Drug Application (sNDA) for Setmelanotide has been accepted by the FDA. The application aims to use Setmelanotide to treat acquired hypothalamic obesity-related diseases. Notably, the FDA has granted this application priority review status and set the Prescription Drug User Fee Act (PDUFA) target review date as December 20, 2025.
Coincidentally, in the European market, the EMA has also confirmed the validation of Rhythm Pharmaceuticals' submitted Type II variation MAA application, which targets the same indication as the aforementioned sNDA. Since August 16, 2025, this application has been under review by the CHMP. Upon completion of the review, the CHMP will provide an opinion to the EC regarding potential approval.
Setmelanotide, as an MC4R agonist, is not new to the medical field. Previously, it has been approved by the FDA, EMA, and the UK MHRA for the treatment of obesity caused by Bardet-Biedl syndrome (BBS) and defects in POMC, PCSK1, or LEPR.
Behind this sNDA and Type II variation application lies solid support from the TRANSCEND Phase III pivotal trial data. This global, randomized, double-blind, placebo-controlled trial enrolled 120 patients with acquired hypothalamic obesity, primarily aiming to evaluate the efficacy and safety of Setmelanotide. The primary endpoint was the change in body mass index (BMI) after 52 weeks of treatment, while secondary endpoints covered daily hunger, binge eating behavior, weight, quality of life, and safety.
The trial results were quite impressive. The group treated with Setmelanotide experienced an average BMI reduction of 16.5%, whereas the placebo group’s BMI increased by an average of 3.3%, with the difference between the two groups being statistically significant (p<0.0001). Further breakdown showed that adult patients had a BMI reduction of 19.2%, and pediatric patients had an even higher reduction of 20.2%. Meanwhile, the drug was generally well tolerated, with the most common adverse events including nausea, vomiting, diarrhea, injection site reactions, skin pigmentation, and headache.
CongenPharma committed to providing green, efficient, and cost-effective peptide drugs, commented: This series of developments undoubtedly brings new treatment hope to patients with acquired hypothalamic obesity and fills people with high expectations for the application prospects of Setmelanotide in this field.
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