European Commission decision anticipated in July
BOSTON, May 21, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending marketing authorization for setmelanotide for the treatment of obesity and the control of hunger associated with 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.
“People living with these rare genetic diseases experience early-onset, rapid weight gain and severe, insatiable hunger, and, despite significant effort to control their weight and appetite with supportive care and lifestyle interventions, patients often regain weight after any short-term period of weight loss,” said Karine Clément, M.D., Ph.D., Professor of Nutrition at Pitié-Salpêtrière Hospital, Sorbonne Université and Head of the INSERM Nutriomics Laboratory in Paris. “With this positive opinion based on data from the largest clinical trials to date in these patient populations, people living with POMC, PCSK1 or LEPR deficiency obesities in Europe may soon have a therapeutic option with the potential to reduce their hunger and body weight.”
The CHMP opinion on setmelanotide, which has the PRIority MEdicines (PRIME) designation, will now be reviewed by the European Commission. A final decision on the marketing authorization application for setmelanotide is anticipated in July 2021. If approved by the European Commission, setmelanotide would be the first treatment in the EU indicated for these rare genetic diseases of obesity and would be commercialized under the brand name IMCIVREE™.
“We are excited by this positive opinion from the CHMP, which marks a significant step toward bringing setmelanotide to patients suffering from the severe obesity and insatiable hunger associated with rare genetic diseases of obesity around the world,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We look forward to the European Commission’s decision in the coming months, as we continue to build our global infrastructure to support commercial availability and broaden our ongoing clinical development program to explore setmelanotide’s potential in other rare genetic diseases of obesity.”
Pivotal Trials and Data Supporting Positive OpinionPOMC, PCSK1 or LEPR deficiency obesities are ultra-rare diseases caused by variants in POMC, PCSK1 or LEPR genes that impair the MC4 receptor pathway, which is a pathway in the hypothalamus that regulates hunger, energy expenditure and, consequently, body weight. People living with obesity due to POMC, PCSK1 or LEPR deficiency struggle with extreme, insatiable hunger beginning at a young age, resulting in early-onset, severe obesity. As an MC4 receptor agonist, setmelanotide is designed to restore impaired MC4 receptor pathway activity arising due to genetic deficits upstream of the MC4 receptor.
The CHMP based its positive opinion on the results from two pivotal trials in which setmelanotide achieved statistically significant and clinically meaningful weight loss and reduction in hunger in patients with POMC, including PCSK1, and LEPR deficiency obesity. Across both studies, statistically significant and clinically meaningful reductions in body mass index (BMI) for adults and BMI-Z scores, which represent the number of standard deviations from median BMI by child age and sex, were achieved. These two studies, which are the largest studies conducted in these disease states to date, were identically designed one-year, open-label studies, each with a double-blind, placebo-controlled withdrawal period. Additional supportive data were gathered in an investigator-led study and an ongoing extension study.
Pivotal Data in POMC, including PCSK1, Deficiency Obesity
- Eight of 10 patients with POMC deficiency obesity achieved the primary endpoint of greater than 10 percent weight loss over approximately one year (p