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Rhythm Pharmaceuticals Receives Orphan Drug Designation from U.S. FDA for Setmelanotide for the Treatment of Alström Syndrome
BOSTON, March 18, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a late-stage biopharmaceutical company aimed at developing and
About this update from Rhythm Pharmaceuticals, Inc.
[{"type":"text","content":"BOSTON, March 18, 2020 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to setmelanotide for the treatment Alström syndrome. People living with Alström syndrome may experience an insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life. Rhythm is currently evaluating setmelanotide’s ability to reduce hunger and affect weight loss in an ongoing pivotal Phase 3 trial in patients living with Alström and Bardet-Biedl syndromes.\n “Individuals and families living with Alström syndrome face a high disease burden, which often adversely affects their daily lives, and yet there are currently no treatment options available to address this serious unmet need,” said Murray Stewart, M.D., Chief Medical Officer of Rhythm. “Orphan drug designation from the FDA reinforces the urgency of our work with setmelanotide in Alström syndrome, as we advance our pivotal Phase 3 trial to topline data expected by the end of this year or early next year.” Orphan drug designation is granted by the FDA to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. About Alström SyndromeAlström syndrome is an ultra-rare genetic disorder that affects multiple organ systems. Insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life may be seen in individuals living with Alström syndrome. Clinical features of Alström syndrome may include progressive visual and auditory impairments, insulin resistance and Type 2 diabetes, hyperlipidemia, progressive kidney dysfunction, cardiomyopathy and short stature in adulthood. There is great variability in presentation and severity of these symptoms across individuals with Alström syndrome. In the United States, the Company estimates that Alström syndrome affects approximately 500 people. Currently, there are no approved therapies t...