Monday, March 27, 2023
Rhythm Pharmaceuticals, Inc., a 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 the publication of interview-based patient and caregiver reported experiences of hunger and quality of life with setmelanotide treatment in Bardet-Biedl syndrome (BBS). The research is published in the peer-reviewed journal Advances in Therapy.
“Hyperphagia and early-onset, severe obesity are hallmark characteristics of BBS that place a considerable burden on patients and their families,” said Prof. Andrea M. Haqq, M.D., Department of Pediatrics, Faculty of Medicine & Dentistry, University of Alberta. “Our results show that hyperphagia – a pathological, all-consuming hunger leading to an obsessive focus on food – negatively affects the lives of patients and families, posing difficulties with concentration, emotional and physical manifestation, and impaired relationships. Our analyses also demonstrate that setmelanotide therapy resulted in meaningful improvements for these patients, such as weight loss and a decrease in obsessive focus on food and food-seeking behaviors. Further, these interviews exemplify the usefulness of patient experience data as recommended by the U.S. Food and Drug Administration for patient-centered drug development.”
The qualitative study publication, titled, “Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life With Setmelanotide Treatment in Bardet-Biedl Syndrome,” includes patients with BBS or their caregivers who participated in Phase 2 and 3 clinical trials of setmelanotide. A total of 19 interviews were conducted with patients (n=8) and caregivers (n=11) to explore patient experience and caregiver observations of hyperphagia before and during setmelanotide treatment. Before setmelanotide treatment, most patients (n=7; 87.5%) and caregivers (n=10; 90.9%) experienced negative effects directly related to hyperphagia. In addition, most participants (15 of 19 overall [78.9%]; 5 of 8 patients [62.5%] and 10 of 11 caregivers [90.9%]) described a lack of control with eating.
All participants reported substantial improvements in hyperphagia and satiety as well as weight loss after initiating setmelanotide treatment, including improved focus and concentration related to reductions in obsessive behaviors associated with food. All caregivers noted the ability to give their child greater autonomy around food choices and consumption and a reduced need to monitor their child’s food intake. Approximately half of patients and caregivers noted that improvements in hunger had benefited family dynamics.
“Until now, the impact of hyperphagia on the lives of these patients and their families has not been fully understood. This study confirms the substantial burden and provides additional insights into how treatment with setmelanotide facilitated improvements in hyperphagia and promoted beneficial changes in patient and caregiver experiences,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.
Rhythm also announced the publication of a study, titled, “Health State Utilities Associated With Hyperphagia: Data for Use in Cost-Utility Models,” that evaluated the assessment of the substantial impact of severe hyperphagia on patients’ quality of life. This study is the first to estimate the impact of hyperphagia on health state utilities independently of any specific underlying indication. These data can be incorporated into cost-utility models conducted to assess the value of treatments for rare MC4R pathway diseases. Data published in the open-access journal Obesity Science and Practice show that increasing severity of hyperphagia is associated with profound impacts on quality of life that are comparable to other severe health states, such as stroke and progressive metastatic cancers, which similarly have a broad impact on many aspects of quality of life.