University of Melbourne Faculty of Medicine, Dentistry and Health Sciences

Rhythm Pharmaceuticals Receives Positive CADTH Reimbursement Recommendation for IMCIVREE® (setmelanotide)

Retrieved on: 
Thursday, November 2, 2023
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BOSTON, Nov. 02, 2023 (GLOBE NEWSWIRE) -- 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).

Key Points: 
  • 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.
  • “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.

Rhythm Pharmaceuticals Announces Two Publications Detailing Burden of Hyperphagia and Obesity on Patients and Caregivers Living with Bardet-Biedl Syndrome

Retrieved on: 
Wednesday, July 19, 2023
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“These results demonstrate that hyperphagia and severe obesity associated with BBS negatively affect performance in school, work and social relationships.

Key Points: 
  • “These results demonstrate that hyperphagia and severe obesity associated with BBS negatively affect performance in school, work and social relationships.
  • Caregivers of patients with BBS reported that hyperphagia has broad impacts, well beyond contributing to obesity.
  • Hyperphagia had at least a moderate negative impact on most patients’ mood/emotions (56%), sleep (54%), school (57%), leisure (62%), and familial relationships (51%).
  • “ Caregiver Burden in Bardet-Biedl Syndrome: Findings from the CARE-BBS Study ,” quantifies caregiver burden associated with obesity and hyperphagia.

Rhythm Pharmaceuticals Announces Health Canada Approval of IMCIVREE® Setmelanotide Injection for Weight Management in Bardet-Biedl Syndrome or Genetically-confirmed Biallelic POMC, PCSK1, or LEPR Deficiency

Retrieved on: 
Monday, May 8, 2023
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BOSTON, May 08, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company, today announced that Health Canada has approved IMCIVREE® (setmelanotide solution for subcutaneous injection) for weight management in adult and pediatric patients 6 years of age and older with 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. Please consult the IMCIVREE product monograph available at www.rhythmtx.ca.

Key Points: 
  • Setmelanotide has been shown to reduce the weight of patients living with BBS or POMC, PCSK1 or LEPR deficiency.
  • The trials met all primary endpoints and key secondary endpoints among patients at 52 weeks on therapy.
  • Results from these trials were featured in several publications, including the peer-reviewed journal The Lancet Diabetes and Endocrinology .
  • The most common adverse reactions were skin hyperpigmentation, injection site reactions and nausea.

Rhythm Pharmaceuticals Announces Publication of Bardet-Biedl Syndrome Patient and Caregiver Perspectives of Hunger and Quality of Life with Setmelanotide

Retrieved on: 
Monday, March 27, 2023
Polyphagia, Faculty, University of Alberta, Obesity, Food, University, Use, Phase II, Stroke, Weight loss, Bardet–Biedl syndrome, Eating, Quality of life, Advances in Therapy, Research, BBS, Patient, Child, MC4R, University of Melbourne Faculty of Medicine, Dentistry and Health Sciences, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Caregiver, Medicine, Pharmaceutical industry, Residential care, Rhythm, Syndrome, Hunger

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.

Key Points: 
  • 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.
  • “Until now, the impact of hyperphagia on the lives of these patients and their families has not been fully understood.
  • These data can be incorporated into cost-utility models conducted to assess the value of treatments for rare MC4R pathway diseases.

Rhythm Pharmaceuticals Announces Publication of Analysis of Quality of Life Improvements from Phase 3 Clinical Trial in Bardet-Biedl Syndrome

Retrieved on: 
Monday, January 23, 2023
Degenerative disease, Very early onset inflammatory bowel disease, Body mass index, Impact, Polyphagia, BBS, Orphanet Journal of Rare Diseases, Human, Bardet–Biedl syndrome, Quality of life, Medicine, University of Melbourne Faculty of Medicine, Dentistry and Health Sciences, Patient, Obesity, University of Alberta, FDA, Hope, Rhythm, Precision medicine, Faculty, Life, Adolescence, University, Pharmaceutical industry, MC4R, HRQOL

BOSTON, Jan. 23, 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 that patients with Bardet-Biedl syndrome (BBS) and obesity who participated in the company’s global Phase 3 clinical trial of setmelanotide reported clinically meaningful improvements across multiple health-related quality of life (HRQOL) measures based on an analysis published in the Orphanet Journal of Rare Diseases.

Key Points: 
  • “Living with hyperphagia and severe obesity associated with BBS presents a clinically significant burden on patients and families,” said corresponding author Prof. Andrea M. Haqq, M.D., Department of Pediatrics, Faculty of Medicine & Dentistry, University of Alberta.
  • For the 52-week analysis, 9 patients younger than 18 years, or their caregiver, reported a significant and clinically meaningful change in their quality of life with a mean change from baseline of +11.2 points on the Pediatric Quality of Life Inventory (PedsQL) measure.
  • In addition, 11 adult patients who completed the Impact of Weight on Quality of Life Questionnaire-Lite (IWQOL-Lite) achieved a significant and clinically meaningful improvement in their weight related quality of life with a mean change from baseline of +12.0 points.
  • IWQOL-Lite score was significantly correlated with changes in percent body weight (P=0.0037) and body mass index (P=0.0098).

Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trial of IMCIVREE® (setmelanotide) in Bardet-Biedl Syndrome in The Lancet Diabetes and Endocrinology

Retrieved on: 
Tuesday, November 8, 2022
People, GLOBE, Polyphagia, BBS, Government budget balance, Adult, MC4R, Visual impairment, SD, Weight, University of Alberta, Nasdaq, Hypogonadism, Bardet–Biedl syndrome, Department, BMI, Faculty, Very early onset inflammatory bowel disease, Obesity, Hunger, Patient, Safety, Weight loss, University of Melbourne Faculty of Medicine, Dentistry and Health Sciences, University, Life, The Lancet, Polydactyly, Medicine, Pharmaceutical industry, Endocrinology, EU

BOSTON, Nov. 08, 2022 (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 that previously disclosed results from a Phase 3 clinical trial that evaluated IMCIVREE® (setmelanotide), an MC4R agonist, in patients with Bardet-Biedl syndrome (BBS) have been published in the peer-reviewed journal The Lancet Diabetes and Endocrinology. The trial met its primary endpoint and all key secondary endpoints, with statistically significant reductions in weight and hunger at 52 weeks on therapy.

Key Points: 
  • People living with BBS may experience insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life.
  • Rhythms multicenter Phase 3 trial (NCT03746522) enrolled patients 6 years old with obesity and BBS or Alstrm syndrome (N=38).
  • The primary endpoint was the proportion of patients 12 years old in the full analysis set achieving 10% weight reduction after 52 weeks.
  • As previously disclosed, treatment with setmelanotide resulted in significant weight and hunger reductions after one year of treatment among patients with BBS.

BioAlberta Recognizes Alberta Innovators and Entrepreneurs at Annual Health and Life Sciences Showcase & Awards Ceremony

Retrieved on: 
Wednesday, September 28, 2022
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The awards were presented at BioAlbertas Annual Health and Life Sciences Showcase & Awards Ceremony, held in Calgary on September 27th.

Key Points: 
  • The awards were presented at BioAlbertas Annual Health and Life Sciences Showcase & Awards Ceremony, held in Calgary on September 27th.
  • This award acknowledges a company that has shown significant achievement within the marketplace and Albertas business community through strong performance or a leadership role.
  • Accepting this award Co-Founders Matt Anderson-Baron and Jalene Anderson-Baron said: We are very honored to be recognized as the BioAlberta company of the year.
  • BioAlberta is the voice and champion for life sciences in Alberta, committed to creating a thriving and competitive industry by facilitating and accelerating economic diversification, investment attraction and job growth.