{"id":770901,"date":"2023-07-19T10:01:40","date_gmt":"2023-07-19T14:01:40","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/rhythm-pharmaceuticals-announces-two-publications-detailing-burden-of-hyperphagia-and-obesity-on-patients-and-caregivers-living-with-bardet-biedl-syndrome\/"},"modified":"2023-07-19T10:01:40","modified_gmt":"2023-07-19T14:01:40","slug":"rhythm-pharmaceuticals-announces-two-publications-detailing-burden-of-hyperphagia-and-obesity-on-patients-and-caregivers-living-with-bardet-biedl-syndrome","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/rhythm-pharmaceuticals-announces-two-publications-detailing-burden-of-hyperphagia-and-obesity-on-patients-and-caregivers-living-with-bardet-biedl-syndrome\/","title":{"rendered":"Rhythm Pharmaceuticals Announces Two Publications Detailing Burden of Hyperphagia and Obesity on Patients and Caregivers Living with Bardet-Biedl Syndrome"},"content":{"rendered":"

\nResults of The CAREgiver Burden in BBS (CARE-BBS) study show multifaceted burden of hyperphagia
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BOSTON, July 19, 2023 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc.\u00a0(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 two new publications detailing the burden of hyperphagia and obesity for adult caregivers, families and patients living with Bardet-Biedl Syndrome (BBS) based on results of The CAREgiver Burden in BBS (CARE-BBS) study. The research is published in the peer-reviewed journal, The Orphanet Journal of Rare Diseases.<\/em><\/p>\n

\u201cBBS is characterized by hyperphagia \u2013 an insatiable hunger that results in extreme food seeking behaviors \u2013 and early-onset, severe obesity, both of which have broad negative impacts on the lives of patients living with BBS and their caregivers,\u201d said Prof. Andrea M. Haqq, M.D., Department of Pediatrics, Faculty of Medicine & Dentistry, University of Alberta. \u201cThese results demonstrate that hyperphagia and severe obesity associated with BBS negatively affect performance in school, work and social relationships. Importantly, the findings from the CARE-BBS study were similar across countries, demonstrating the universality of hyperphagia and underscoring the need for therapies that can effectively treat hyperphagia in order to alleviate the extensive clinical and nonclinical impacts of the disease.\u201d<\/p>\n

\u201cBurden of Hyperphagia and Obesity in Bardet-Biedl Syndrome: a Multi-country Survey,<\/a>\u201d includes results from a cross-sectional survey of 242 adult caregivers of patients with BBS across the United States, the United Kingdom, Canada and Germany. Caregivers of patients with BBS reported that hyperphagia has broad impacts, well beyond contributing to obesity. Caregivers observed hyperphagic behaviors throughout the day, most frequently negotiating for food (90%) and waking up and asking or looking for food during the night (88%). Hyperphagia had at least a moderate negative impact on most patients\u2019 mood\/emotions (56%), sleep (54%), school (57%), leisure (62%), and familial relationships (51%).<\/p>\n

\u201cCaregiver Burden in Bardet-Biedl Syndrome: Findings from the CARE-BBS Study<\/a>,\u201d quantifies caregiver burden associated with obesity and hyperphagia. The findings show that patients\u2019 hyperphagia had a moderate-to-severe impact on caregiver mood (56.6%), sleep (46.6%), and relationships (48.0%), contributing to a high level of personal strain and work productivity loss for caregivers in the workforce, as well as a meaningful family impact.<\/p>\n

\u201cWe continue to learn more about the full extent of hyperphagia\u2019s negative impact on BBS patients, their families and caregivers,\u201d said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. \u201cThese results provide valuable insights into the physical, social and economic burdens of hyperphagia for both patients and their families and show the need for therapies designed to alleviate hyperphagia and, as a result, improve quality of life.\u201d<\/p>\n

\n About Bardet-Biedl Syndrome
<\/strong>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>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) is approved by the\u00a0U.S. Food and Drug Administration\u00a0(FDA) and authorized by the European Commission\u00a0(EC) and the UK\u2019s\u00a0Medicines & Healthcare Products Regulatory Agency\u00a0(MHRA) for use in accordance with product labeling. 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\u00a0Boston, 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 proopiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.<\/p>\n

In\u00a0Canada, setmelanotide is indicated for the treatment of obesity due to Bardet-Biedl syndrome (BBS) or genetically-confirmed biallelic pro-opiomelanocortin (POMC), proprotein convertase subtilisin\/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency due to variants interpreted as pathogenic, likely pathogenic, or of uncertain significance in adults and children 6 years of age and above.<\/p>\n

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