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Rhythm Pharmaceuticals Announces FDA Acceptance for Filing and Priority Review of Supplemental New Drug Application for IMCIVREE® (setmelanotide) for Patients with Bardet-Biedl Syndrome and Alström Syndrome
FDA sets PDUFA goal date of March 16, 2022 BOSTON, Nov. 15, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"FDA sets PDUFA goal date of March 16, 2022\nBOSTON, Nov. 15, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the company’s supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide), a melanocortin-4 receptor (MC4R) agonist, for patients with Bardet-Biedl syndrome (BBS) or Alström syndrome. The FDA granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 16, 2022. “The acceptance for filing of our supplemental application by the FDA marks an important step in our efforts to address the unmet needs and bring IMCIVREE to patients and families living with Bardet-Biedl syndrome and Alström syndrome,” said Linda Shapiro, M.D., Ph.D., Chief Medical Officer of Rhythm Pharmaceuticals. “The FDA’s decision to grant Priority Review to the application aligns with our belief in the potential of IMCIVREE to deliver clinically meaningful and statistically significant reductions in body weight and hunger for patients with BBS and Alström syndrome while also substantially improving quality of life for these patients and their families.” A Priority Review designation is granted by the FDA for the evaluation of drug applications that may offer significant improvements in the safety or effectiveness of the treatment, prevention or diagnosis of a serious disease. Under the FDA’s current PDUFA review goals, for an application granted Priority Review, the FDA aims to take action on such application within six months of receipt, compared to 10 months under standard review. About Bardet-Biedl and Alström SyndromesBBS and Alström syndrome are ultra-rare genetic diseases that affect multiple organ systems. Clinical features of BBS may include early-onset severe obesity, hyperphagia, cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and/or visual impairment. Clinical features of Alström syndrome may include early-onset severe obesity, hyperphagia, progressive visual and auditory impairment, insulin resistance and Type 2 diabetes, hyperlipidemia, progressive kidney dysfunction, cardiomyopathy, and short stature in adulthood. Hyperphagia ...