Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended that IMCIVREE® (setmelanotide) be reimbursed by CADTH-participating public drug plans, with conditions, for weight management in adult and pediatric patients six years of age and older with obesity due to Bardet-Biedl syndrome (BBS).
“This positive CADTH recommendation will be welcome news for the patients and families living with hyperphagia, which is a pathological and insatiable hunger, and the severe obesity that is often associated with BBS,” said Prof. Andrea M. Haqq, M.D., Department of Pediatrics, Faculty of Medicine & Dentistry, University of Alberta. “This important recommendation further recognizes the unmet needs of people with BBS who otherwise have no targeted precision treatment for weight management.”
CADTH’s recommendation is based on results from Rhythm’s Phase 3 trial that demonstrated treatment with setmelanotide resulted in meaningful clinical benefit in patients 6 years of age and older with obesity due to BBS. Data showed that treatment with setmelanotide for 52 weeks resulted in a clinically meaningful reduction in weight-related parameters, such as total body weight and body mass index (BMI), and the results were supported by the exploratory comparative 14-week placebo-controlled outcomes. Consistent with prior clinical experience, setmelanotide was generally well tolerated. Treatment-emergent adverse events (TEAEs) included mild injection site reactions and nausea.
BBS is a rare genetic disease with an estimated prevalence of 1 in 100,000 to 160,000 in the populations of North America and Europe with an estimated 300-400 people living with the disease in Canada. People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life. BBS may also be associated with cognitive impairment, polydactyly, renal dysfunction, hypogonadism, and visual impairment.
IMCIVREE® (setmelanotide) is the first and only therapy approved in Canada for weight management in adult and pediatric patients 6 years of age and older with obesity due to BBS. IMCIVREE® (setmelanotide) was given a Priority Review by Health Canada.
“This recommendation represents a significant milestone for Canadian patients living with obesity due to BBS. We appreciate CADTH’s recommendation and their recognition of the rare genetic nature of BBS, the unmet needs of these patients and that IMCIVREE is the only available therapy that targets the underlying disease mechanism of BBS,” said Carol Stiff, General Manager of Rhythm Pharmaceuticals Canada Inc.