Rhythm Receives FDA Approval for Imcivree in Certain Patients with Obesity

 

Rhythm Pharmaceuticals announced on Nov. 27 that the U.S. Food and Drug Administration (FDA) has approved of IMCIVREE (setmelanotide) for chronic weight management in adult and pediatric patients aged six 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 product is now the first-ever FDA approved therapy for these genetic diseases linked to obesity. The FDA approval was based on results from the largest studies conducted to date in obesity associated with POMC, PCSK1 or LEPR deficiency.

According to data from Phase III clinical trials, approximately 80% of patients with obesity due to POMC or PCSK1 deficiency achieved greater than 10% weight loss. A total of 45.5% of patients with obesity due to LEPR deficiency achieved greater than 10% weight loss after one year of treatment with IMCIVREE.

“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,” said David Meeker, M.D., Chair, president and chief executive officer of Rhythm. “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.”

These types of 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. The pathway is located in the hypothalamus and is responsible for regulating hunger, energy expenditure and ultimately body weight.

“Many patients and families who live with these diseases face an often burdensome stigma associated with severe obesity,” said Jennifer Miller, M.D., pediatric endocrinologist at University of Florida Health. “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.”

Back in July, Rhythm Pharmaceuticals announced that the FDA had granted its rare pediatric disease designations for setmelanotide. Under this designation, the FDA could grant a priority review voucher to a sponsor who receives a product approval for a “rare pediatric disease,” defined as a serious or life-threatening disease.

“We believe that receipt of the rare pediatric disease designation for setmelanotide, for which we have previously received Breakthrough Therapy and orphan drug status, underscores the FDA’s recognition of setmelanotide’s potential to treat POMC and LEPR deficiency obesities; two serious, ultra-rare conditions for which existing treatment options are woefully inadequate,” said Murray Stewart, M.D., Chief Medical Officer of Rhythm, at the time of the announcement. “As a result of early-onset, severe obesity and insatiable hunger, many patients experience debilitating comorbidities beginning in childhood, which worsen over time and can greatly affect their quality of life. With setmelanotide, we believe we have the potential to modify this disease trajectory by delivering a therapy that can be dosed chronically beginning in childhood. We are grateful to the FDA for this designation and look forward to continuing to work toward our goal of delivering setmelanotide to people with rare genetic disorders of obesity.”

Setmelanotide is currently being developed by Rhythm as a targeted therapy to potentially restore the function of an impaired MC4R pathway in patients.