Setmelanotide significantly reduces body weight for people with Bardet-Biedl syndrome
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Setmelanotide led to significant reductions in weight among people with obesity and the genetic disorder Bardet-Biedl syndrome, but not Alström syndrome, according to findings published in The Lancet Diabetes & Endocrinology.
“Hyperphagia, which is a pathological hunger, and early-onset, severe obesity place a significant burden on both patients living with [Bardet-Biedl syndrome] and their families,” Andrea M. Haqq, MD, professor in the department of pediatrics and the division of pediatric endocrinology/metabolism in the faculty of medicine and dentistry at the University of Alberta in Canada, said in a related press release. “The results from this pivotal phase 3 clinical trial — the largest and longest interventional study ever conducted in Bardet-Biedl syndrome — showed patients with Bardet-Biedl syndrome achieved clinically significant weight loss, reductions in hunger and improvements in health-related quality of life, all of which are highly meaningful to patients with Bardet-Biedl syndrome and their caregivers.”
This multicenter, randomized, 14-week, double-blind, placebo-controlled, phase 3 clinical trial enrolled 38 participants from 12 sites between December 2018 and November 2019 with a clinical diagnosis of Bardet-Biedl syndrome (n = 32) or Alström syndrome (n = 6) and obesity. All participants were randomly assigned to subcutaneous setmelanotide 3 mg (Imcivree, Rhythm Pharmaceuticals; n = 19) or placebo (n = 19) once per day over 14 weeks followed by setmelanotide for 52 weeks.
The primary outcome was the proportion of participants aged 12 years or older who reached at least 10% body weight reduction from baseline at 52 weeks of setmelanotide treatment.
Overall, 32.3% of participants aged 12 years or older with Bardet-Biedl syndrome experienced at least 10% body weight reduction with setmelanotide treatment for 52 weeks (P = .0006). Researchers observed a mean BMI reduction of –9.1% among 15 participants aged 18 years or older, a change in BMI z score of –0.8 among 14 participants younger than 18 years and a BMI z score change of –0.2 points or more among 12 of these participants. In addition, 14 participants aged 12 years or older experienced a reduction of –30.5% in their maximal hunger scores.
Regarding adverse outcomes, 61% of participants reported skin hyperpigmentation and 48% reported injection-site erythema. However, two participants experienced four serious adverse outcomes, including blindness, anaphylactic reaction and suicidal ideation, but none were related to the study drug, according to the researchers.
“With early, significant and sustained weight reduction demonstrated in our phase 3 trial, we were able to secure approvals in the United States and European Union and deliver the first treatment for obesity in patients with Bardet-Biedl syndrome,” David Meeker, MD, chair, president and CEO of Rhythm Pharmaceuticals, said in the release. “We look forward to leveraging the publication of these trial results to drive continued momentum, as we work toward our goal of bringing a new option to people who are living with this rare genetic disease around the world, while also targeting additional rare [melanocortin-4 receptor] pathway diseases with our broad ongoing clinical development program.”
Based on data from this phase 3 trial, setmelanotide has been approved as the first therapy for chronic weight management for adults and children aged 6 years or older with Bardet-Biedl syndrome in the U.S. and European Union.
Reference:
- Rhythm Pharmaceuticals announces publication of results from phase 3 clinical trial of Imcivree (setmelanotide) in Bardet-Biedl syndrome in The Lancet Diabetes and Endocrinology. Published Nov. 7, 2022. Accessed Nov. 8, 2022.
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