The drug is the first FDA-approved therapy for these rare genetic diseases.
FDA cleared the first treatment for rare genetic diseases of obesity.
The agency approved setmelanotide (Imcivree, Rhythm Pharmaceuticals) injection for chronic weight management in adults 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.
“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.” said David Meeker, MD, chair, president and CEO of Rhythm, in a press release.
Rhythm expects the treatment to available in the first quarter of 2021.
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 — a pathway in the hypothalamus that is responsible for regulating hunger, energy expenditure and consequently body weight, Rhythm explained.
“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,” said Jennifer Miller, ND, pediatric endocrinologist at University of Florida Health.
“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,” Miller added.
The FDA approval is based on Phase 3 clinical trials in which 80% of patients with obesity due to POMC or PCSK1 deficiency achieved greater than 10% weight loss. In addition, 45.5% of patients with obesity due to LEPR deficiency achieved greater than 10% weight loss after one year of treatment with Imcivree.