Biopharmaceutical company Rhythm Pharmaceuticals Inc. (RYTM:NASDAQ), which is focused on developing treatments for rare genetic obesity diseases, today announced that "the U.S. Food & Drug Administration (FDA) has approved IMCIVREE™ (setmelanotide) for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing." The firm noted that this approval of IMCIVREE by the FDA represents the first-ever approved therapy for these rare genetic obesity diseases by the agency.
Rhythm Pharmaceuticals' Chair, President and CEO David Meeker, M.D., commented, "Our first new drug approval is a major milestone for Rhythm, and we look forward to delivering on the promise of IMCIVREE for patients suffering with obesity due to POMC, PCSK1 or LEPR deficiency...With IMCIVREE, we are advancing a first-in-class, precision medicine that is designed to directly address the underlying cause of obesities driven by genetic deficits in the melanocortin-4 (MC4) receptor pathway."
The company explained that "obesity due to POMC, PCSK1 or LEPR deficiency 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 is responsible for regulating hunger, energy expenditure and consequently body weight."
The firm added that those individuals living with obesity due to these medical conditions struggle with extreme, insatiable hunger, which starts a very young age leading to early-onset, severe obesity.
The company noted that its IMCIVREE is an MC4 receptor agonist which is formulated to restore impaired MC4 receptor pathway activity caused by certain genetic deficits. Rhythm Pharmaceuticals indicated that until now there have been no FDA-approved medications for weight management in obesity due to POMC, PCSK1 or LEPR deficiency. The company stated that it is planning to roll out IMCIVREE in the U.S. for commercial use in Q1/21.
Jennifer Miller, M.D., pediatric endocrinologist at University of Florida Health, remarked, "Many patients and families who live with these diseases face an often burdensome stigma associated with severe obesity. To manage this obesity and control disruptive food-seeking behavior, caregivers often lock cabinets and refrigerators and significantly limit social activities...This FDA approval marks an important turning point, providing a much needed therapy and supporting the use of genetic testing to identify and properly diagnose patients with these rare genetic diseases of obesity."
The firm stated that "in Phase 3 clinical trials, 80% of patients with obesity due to POMC or PCSK1 deficiency achieved greater than 10% weight loss and 45.5% of patients with obesity due to LEPR deficiency achieved greater than 10% weight loss after 1 year of treatment with IMCIVREE."
The company's Chief Medical Officer Murray Stewart, M.D., added, "We know that not all obesity is the same, and genetic testing plays a key role in enabling physicians, patients and families to understand the underlying cause of certain severe obesities...In addition to POMC, PCSK1 and LEPR genes, we are continuing our efforts to identify further genes and populations to evaluate the potential for setmelanotide to address the insatiable hunger and early-onset severe obesity that characterize these diseases."
The firm pointed out that the FDA has issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Rhythm that can be redeemed to receive priority review for any subsequent marketing application. The voucher may also be licensed, transferred or sold at Rhythm's discretion.
The company stated that it is also now evaluating setmelanotide in another Phase 3 trial for reduction in hunger and body weight in people with Bardet-Biedl or Alström syndrome. The firm said it expects to receive the topline data from this trial in Q1/21 at the latest.
Rhythm Pharmaceuticals is a biopharmaceutical company headquartered in Boston, Mass. The firm specializes in creating and commercializing therapies for treating rare genetic diseases of obesity. The company listed that "it is leveraging the Rhythm Engine and the largest known obesity DNA database, now with more than 30,0000 sequencing samples from individuals with severe obesity, to improve the understanding, diagnosis and potentially the treatment of rare genetic diseases of obesity."
Rhythm Pharmaceuticals started the day with a market capitalization of around $1.1 billion with approximately 44.2 million shares outstanding and a short interest of about 4.3%. RYTM shares opened nearly 13% higher today at $27.22 (+$3.06, +12.67%) over the prior trading day's $24.16 closing price. The stock has traded today between $26.12 and $31.24 per share and is currently trading at $30.40 (+$6.24, +25.83%).[NLINSERT]
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