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Rhythm Pharmaceuticals Announces FDA Acceptance for Priority Review of Supplemental New Drug Application for IMCIVREE® (setmelanotide) in Patients as Young as 2 years old
-- Label expansion sought to treat younger children with Bardet-Biedl syndrome or POMC/LEPR deficiency -- -- FDA sets PDUFA goal date of December 26, 2024 --
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"-- Label expansion sought to treat younger children with Bardet-Biedl syndrome or POMC/LEPR deficiency -- -- FDA sets PDUFA goal date of December 26, 2024 -- BOSTON, Aug. 26, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide), a melanocortin-4 receptor (MC4R) agonist, for the treatment of obesity due to Bardet-Biedl syndrome (BBS) or pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency obesity in children as young as 2 years old. The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 26, 2024. “This milestone brings us another step closer to offering a precision therapy for young patients in the U.S. experiencing hyperphagia – a pathological hunger that leads to aberrant food-seeking behaviors – and severe obesity caused by rare MC4R pathway diseases,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. “IMCIVREE has been approved for more than two years and prescribed to hundreds of patients 6 years old and older, and we believe that treating patients at an even earlier age will positively affect the lives of these children and their families.” The sNDA submission is based on data from Rhythm’s multi-center, one-year, open-label Phase 3 trial in patients (N=12) between 2 and younger than 6 years old with obesity due to biallelic POMC/PCSK1 or LEPR deficiency or a clinical diagnosis of BBS. The hyperphagia and severe obesity of rare genetically-caused MC4R pathway diseases can present early in life, and these data showed potential efficacy in patients younger than 6 years of age. Treatment with setmelanotide achieved the primary endpoint with a 3.04 mean reduction in BMI-Z score (a measure of body mass index deviations from what is considered normal) and 18.4 percent mean reduction in BMI. In July 2024, the European Commission (EC) expanded the marketing authorization for IMCIVREE to include children as young as 2 years old with obesity ...