Rhythm Pharmaceuticals Announces FDA Acceptance for Priority Review of Supplemental New Drug Application for IMCIVREE® (setmelanotide) in Patients as Young as 2 years old

Core Viewpoint - Rhythm Pharmaceuticals has received FDA acceptance for a supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide) aimed at treating obesity in children as young as 2 years old with Bardet-Biedl syndrome or POMC/LEPR deficiency, with a PDUFA goal date set for December 26, 2024 [1][2] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead product IMCIVREE® designed to treat hyperphagia and severe obesity [4] - The company has been operational for over two years, with IMCIVREE already prescribed to hundreds of patients aged 6 and older [2] Product Information - IMCIVREE is an MC4R agonist approved for chronic weight management in patients aged 6 years and older with specific genetic obesity conditions, including POMC, PCSK1, or LEPR deficiencies, and Bardet-Biedl syndrome [5][6] - The recent sNDA submission is based on a Phase 3 trial involving 12 patients aged 2 to under 6 years, demonstrating a mean reduction of 3.04 in BMI-Z score and an 18.4% mean reduction in BMI [2] Regulatory Developments - The European Commission expanded the marketing authorization for IMCIVREE to include children as young as 2 years old in July 2024, aligning with the recent FDA application [3][6] - The FDA has granted Priority Review status to the sNDA, indicating a faster review process [1]