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Rhythm Pharmaceuticals Announces FDA Extension of Review Period for IMCIVREE® (setmelanotide) for Patients with Bardet-Biedl Syndrome and Alström Syndrome
-- FDA sets updated PDUFA goal date of June 16, 2022 -- -- Company also announces withdrawal of proposed Alström syndrome indication from Type II variation
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"-- FDA sets updated PDUFA goal date of June 16, 2022 -- -- Company also announces withdrawal of proposed Alström syndrome indication from Type II variation application under review by the EMA -- BOSTON, Feb. 24, 2022 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced that the U.S. Food and Drug Administration (FDA) has extended by three months the review period for the supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide) for the treatment of obesity and control of hunger in adult and pediatric patients 6 years of age and older with Bardet-Biedl Syndrome (BBS) or Alström syndrome. On February 23, the FDA notified the Company that the Prescription Drug User Fee Act (PDUFA) goal date has been revised to June 16, 2022. The FDA this month requested additional subgroup analyses of the clinical efficacy data from Rhythm’s Phase 3 pivotal trial in BBS and Alström syndrome. No new data were requested. The additional information has been deemed a ‘major amendment’ to the sNDA, which requires additional time to review. The major amendment did not include any information relating to the safety or manufacturing of setmelanotide. “While we are disappointed by this delay, we believe these additional subgroup analyses are supportive of setmelanotide’s potential to address the early-onset, severe obesity and hyperphagia that characterize both BBS and Alström syndrome,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We recognize patients living with BBS and Alström syndrome have no approved therapies, and we will continue our intense preparations for launch in anticipation of potential regulatory approval.” Withdrawal of Alström syndrome from Type II variation application to EMA for BBSRhythm recently decided to withdraw the proposed Alström syndrome indication from its pending Type II variation application to the European Medicines Agency (EMA) for setmelanotide for the treatment of obesity and control of hunger in adult and pediatric patients 6 years of age and older with BBS. Rhythm made this decision based on feedback from the EMA in order enable the review for BBS within the planned timeline. The Company continues to evaluate ...