FDA expands setmelanotide approval for rare forms of obesity to children aged 2 to 5 years
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Key takeaways:
- The FDA approval for setmelanotide now includes children aged 2 to 5 years with several rare genetic forms of obesity.
- Setmelanotide reduced BMI by 18% at 1 year for children participating in the VENTURE trial.
The FDA approved an expanded indication for setmelanotide to include children aged 2 to 5 years with several genetic forms of obesity, Rhythm Pharmaceuticals announced.
Setmelanotide (Imcivree, Rhythm Pharmaceuticals) is a melanocortin-4 receptor agonist that the FDA previously approved for chronic weight management in people with Bardet-Biedl syndrome in 2022 and those with pro-opiomelanocortin (POMC) deficiency, proprotein convertase subtilisin/kexin type 1 (PCSK1) deficiency or leptin receptor (LEPR) deficiency in 2020. The FDA’s original approvals for setmelanotide were for children and adults aged 6 years and older. The new indication expands the FDA approval to include children aged 2 to 5 years.
The expanded indication follows the publication of findings from the VENTURE phase 3 trial in The Lancet Diabetes & Endocrinology in November. The trial enrolled 12 children aged 2 to 5 years with POMC deficiency, LEPR deficiency or Bardet-Biedl syndrome. All participants received once-daily open-label setmelanotide for 1 year.
At the conclusion of the study, 83% of children had a 0.2-point or greater reduction in BMI z score. Setmelanotide lowered BMI by 18% from baseline to 1 year for the study group. Children with POMC or LEPR deficiency had a mean 26% decrease in BMI at 1 year, and those with Bardet-Biedl syndrome had a mean 10% decline in BMI. Caregivers reported 91% of study participants had declines in hunger over the course of the study.
All adverse events were considered mild or moderate in severity, with no severe adverse events reported. The most common treatment-emergent adverse events were skin hyperpigmentation, vomiting, nasopharyngitis, upper respiratory tract infection, injection site bruising, injection site pruritus, pyrexia, fall and melanocytic naevus.
Ilene Fennoy, MD, MPH, pediatric endocrinologist, obesity specialist and professor of pediatrics at Columbia University Medical Center, and a co-investigator on the VENTURE trial, said the expanded approval will provide health care professionals with a targeted therapy for children who are diagnosed with rare genetic forms of obesity at a young age.
“It’s important to understand that rare melanocortin-4 receptor pathway diseases differ from general obesity as the insatiable hunger these patients experience is pathologic and a result of impairment to a pathway in the brain,” Fennoy said in a press release from Rhythm Pharmaceuticals. “With this insatiable hunger, most patients develop early-onset obesity before the age of 5. Obesity in childhood, if untreated, can lead to a greater risk of severe and long-term health complications, making early intervention to treat obesity critical. With this expanded indication for Imcivree, patients now can receive a much needed, targeted treatment that we believe can address a root cause of their obesity at a very young age.”
Prior studies found setmelanotide can reduce hunger and lower body weight for people with rare genetic forms of obesity. As Healio previously reported, setmelanotide conferred a 10% or greater reduction in body weight among 32.3% of participants with Bardet-Biedl syndrome in a phase 3 trial. Another study published in Advances in Therapy found setmelanotide lowered hyperphagia and improved emotional well-being for people with Bardet-Biedl syndrome within 2 months of setmelanotide initiation.
Tim Ogden, president of the Bardet-Biedl Syndrome Foundation, said the expanded indication will greatly children with Bardet-Biedl syndrome as well as their families.
“Many children with Bardet-Biedl syndrome feel hungry or think about food regardless of how much or how recently they've eaten, leaving families to deal with children sneaking or stealing food, which makes daily life extremely stressful,” Ogden said in the release. “Parents have enough to worry about and manage when their child has a multi-systemic syndrome like Bardet-Biedl syndrome and Imcivree can be an important tool for their obesity."
Reference:
Argente J, et al. Lancet Diabetes Endocrinol. 2024;doi:10.1016/S2213-8587(24)00273-0.
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