Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE™ (setmelanotide) as First-ever Therapy for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency

Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE™ (setmelanotide) as First-ever Therapy for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency

This week, Rhythm Pharmaceuticals, a biopharmaceutical company specializing in the treatment of rare genetic diseases surrounding obesity, announced the U.S. Food & Drug Administration’s (FDA) approval of their new drug, IMCIVREE™(setmelanotide). IMCIVREE is used for chronic weight management in adult and pediatric patients six years of age and older suffering with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency. IMCIVREE will become the first FDA-approved therapy for patients dealing with obesity due to these rare genetic diseases. 

Rhythm Pharmaceutical’s new drug, IMCIVREE, treats the genes responsible for regulating hunger, energy, expenditure and body weight, the MC4 receptor pathway in the hypothalamus. IMCIVREE restores the MC4 receptor pathway, reducing hunger and helping obese patients diagnosed with POMC, PCSK1 or LEPR deficiency to lose weight. Obesity due to POMC, PCSK1 or LEPR is an incredibly rare set of diseases that causes extreme, insatiable hunger at a young age, resulting in early-onset, severe obesity. Prior to IMCIVREE, no FDA-approved therapies were available to treat obesity from POMC, PCSK1 or LEPR deficiency. The drug is expected to be accessible to patients six years of age and older in the first quarter of 2021.

David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm said, “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.”

Jennifer Miller, M.D., pediatric endocrinologist at University of Florida Health said, “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.”

Rhythm Pharmaceuticals’s study of obesity due to POMC, PCSK1 or LEPR deficiency was the most extensive study to date on the topic. In IMCIVREE’s Phase 3 clinical trials, 80 percent of obese patients suffering with POMC or PCSK1 deficiency achieved greater than ten percent weight loss. 45.5 percent of patients with obesity due to LEPR deficiency achieved greater than ten percent weight loss after one year of treatment with IMCIVREE. 

In its trials, IMCIVREE was generally well-tolerated by most patients. Common adverse reactions included injection site reaction, skin hyperpigmentation and nausea. Potential warnings and precautions include disturbance in sexual arousal, depression, suicidal thoughts, skin pigmentation and darkening of pre-existing nevi. There may be a risk of serious adverse reactions due to benzyl alcohol preservative in neonates and low birth weight infants. IMCIVREE is not approved for use in neonates or infants.

IMCIVREE is poised to become available to patients ages six and up starting in the first quarter of 2021. Learn more about IMCIVREE at https://www.rhythmtx.com/IMCIVREE/prescribing-information.pdf