Rhythm Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide) for treating obesity caused by Bardet-Biedl syndrome (BBS) or pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency in children aged 2 to under 6 years. The FDA granted Priority Review and set a Prescription Drug User Fee Act (PDUFA) goal date of December 26, 2024.
David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm, stated, "This milestone brings us another step closer to offering a precision therapy for young patients in the U.S. experiencing hyperphagia and severe obesity caused by rare MC4R pathway diseases."
Clinical Trial Data
The sNDA is based on data from a multi-center, one-year, open-label Phase 3 trial (N=12) involving patients aged 2 to under 6 years with obesity due to biallelic POMC/PCSK1 or LEPR deficiency or a clinical diagnosis of BBS. The study demonstrated that setmelanotide achieved the primary endpoint, with a 3.04 mean reduction in BMI-Z score and an 18.4 percent mean reduction in BMI.
Regulatory Landscape
In July 2024, the European Commission (EC) expanded the marketing authorization for IMCIVREE to include children as young as 2 years old with obesity due to BBS or POMC/PCSK1, or LEPR deficiency. In the United States, IMCIVREE is currently indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS), and BBS.
About Setmelanotide
Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to treat hyperphagia and severe obesity. It is already approved in the U.S. for chronic weight management in patients 6 years and older with specific genetic obesity conditions. The European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have also authorized setmelanotide for treating obesity and controlling hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. The EC has also authorized setmelanotide for control of hunger and treatment of obesity in children as young as 2 years old, living with BBS or POMC, PCSK1, or LEPR deficiency.
