FDA approves setmelanotide for patients with Bardet-Biedl syndrome
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The FDA has cleared a melanocortin-4 receptor agonist for chronic weight management in people with a rare genetic disease of obesity, according to an industry press release.
Setmelanotide (Imcivree, Rhythm Pharmaceuticals) has been approved for chronic weight management in people aged 6 years and older with Bardet-Biedl syndrome, a disease that causes hyperphagia and severe obesity beginning early in life.
Setmelanotide targets impairment in the melanocortin-4 receptor (MC4R) pathway, which is a root cause of early-onset severe obesity and hyperphagia. Setmelanotide was previously approved for people aged 6 years and older with monogenic or syndromic obesity due to proopiomelanocortin, proprotein convertase subtilisin/kexin type 1 or leptin receptor deficiency in November 2020.
“This FDA approval represents a significant milestone for Rhythm, validating our strategy of developing Imcivree for people with hyperphagia and severe obesity caused by rare MC4R-pathway diseases and allowing us to provide our precision therapy to an established community of patients living with [Bardet-Biedl syndrome] and their families who are eagerly awaiting a new treatment option,” David Meeker, MD, chair, president and CEO of Rhythm Pharmaceuticals, said in a press release. “We look forward to delivering this important medicine to the growing community of patients and families in need of options that can effectively address the obesity and hyperphagia that affect many people living with [Bardet-Biedl syndrome].”
In a phase 3 trial, 31 people aged 6 years and older with obesity due to Bardet-Biedl syndrome achieved a 7.9% reduction in BMI with setmelanotide at 1 year without requirements for diet or exercise. In a 14-week, double-blind placebo-controlled stage, participants using setmelanotide had a 4.6% reduction in BMI compared with a 0.1% reduction for placebo. Fourteen participants aged 12 years and older reported a significant decrease in self-hunger at 1 year in the trial.
Setmelanotide was generally well-tolerated in clinical trials. The most common adverse reactions were skin hyperpigmentation, injection site reactions and nausea.
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