{"id":742744,"date":"2023-03-27T08:05:14","date_gmt":"2023-03-27T12:05:14","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/rhythm-pharmaceuticals-announces-publication-of-bardet-biedl-syndrome-patient-and-caregiver-perspectives-of-hunger-and-quality-of-life-with-setmelanotide\/"},"modified":"2023-03-27T08:05:14","modified_gmt":"2023-03-27T12:05:14","slug":"rhythm-pharmaceuticals-announces-publication-of-bardet-biedl-syndrome-patient-and-caregiver-perspectives-of-hunger-and-quality-of-life-with-setmelanotide","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/rhythm-pharmaceuticals-announces-publication-of-bardet-biedl-syndrome-patient-and-caregiver-perspectives-of-hunger-and-quality-of-life-with-setmelanotide\/","title":{"rendered":"Rhythm Pharmaceuticals Announces Publication of Bardet-Biedl Syndrome Patient and Caregiver Perspectives of Hunger and Quality of Life with Setmelanotide"},"content":{"rendered":"
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\n – Interview-based research published in <\/em>Advances in Therapy demonstrates setmelanotide improved hyperphagia and reduced body weight and obsessive focus on food \u2013<\/em><\/p>\n

\n – Company also announces publication of health state utilities study in hyperphagia –<\/em>\n <\/p>\n

BOSTON, March 27, 2023 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), 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<\/em>.<\/p>\n

\u201cHyperphagia and early-onset, severe obesity are hallmark characteristics of BBS that place a considerable burden on patients and their families,\u201d said Prof. Andrea M. Haqq, M.D., Department of Pediatrics, Faculty of Medicine & Dentistry, University of Alberta. \u201cOur results show that hyperphagia \u2013 a pathological, all-consuming hunger leading to an obsessive focus on food \u2013 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.\u201d<\/p>\n

The qualitative study publication, titled, \u201cInterview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life With Setmelanotide Treatment in Bardet-Biedl Syndrome<\/a>,\u201d 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.<\/p>\n

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\u2019s food intake. Approximately half of patients and caregivers noted that improvements in hunger had benefited family dynamics.<\/p>\n

\u201cUntil 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,\u201d said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm.\u00a0<\/p>\n

Rhythm also announced the publication of a study, titled, \u201cHealth State Utilities Associated With Hyperphagia: Data for Use in Cost-Utility Models<\/a>,\u201d that evaluated the assessment of the substantial impact of severe hyperphagia on patients\u2019 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<\/em> 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.<\/p>\n

\n About Bardet-Biedl Syndrome<\/strong>
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Bardet-Biedl syndrome (BBS) is a heterogenous rare genetic syndromic disease that presents with a variety of symptoms that evolve over time including visual impairments, renal disease, polydactyly, genital abnormalities, cognitive impairment, and hyperphagia and early-onset, severe obesity arising from impairment of the hypothalamic MC4R pathway. In the United States, BBS affects approximately 4,000 to 5,000 individuals with similar prevalence in Europe.<\/p>\n

\n About\u00a0Rhythm Pharmaceuticals<\/strong>
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Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) diseases. Rhythm\u2019s lead asset, IMCIVREE (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases, is approved by the U.S. Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin\/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS). Both the European Commission (EC) and the UK\u2019s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare MC4R pathway diseases, as well as a preclinical suite of investigational candidates for the treatment of congenital hyperinsulinism. Rhythm\u2019s headquarters is in Boston, MA.<\/p>\n

\n Setmelanotide Indication<\/strong>
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In\u00a0the\u00a0United States, setmelanotide is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS) or BBS.<\/p>\n

In the\u00a0European Union, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) or genetically confirmed loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.<\/p>\n

\n Limitations of Use<\/strong>
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In\u00a0the United States\u00a0and\u00a0Europe, Setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology.<\/p>\n

Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective:<\/p>\n