The first new drug to treat obesity caused by specific genetic defects has been approved

Recently, Rhythm Pharmaceuticals ("Rhythm Pharmaceuticals") announced that the U.S. FDA has approved its Imcivree™ (setmelanotide) for chronic weight management in adults and children (6 years and older) caused by obesity caused by obesity caused by POMC, pre-protein-converting enzyme oxalolytic 1 (PCSK1) or leptin-infested (LEPR) gene defects. With this approval, Imcivree became the first FDA-approved treatment for obesity from these rare genetic diseases. Previously, the FDA awarded setmelanotide a breakthrough therapy for the treatment of black cortide-4 (MC4) gene defects associated with upper-level gene defects, as well as POMC (including PCSK1) and LEPR defects caused by obesity orphan drug qualification.Imcivree's approval
based on the results of two 52-week Phase 3 clinical trials in obese patients with POMC, PCSK1, or LEPR defects. The results showed that 80 percent of obese patients with POMC or PCSK1 gene defects lost more than 10 percent of their body weight after one year of treatment with Imcivree, and 45.5 percent of obese patients with LEPR defects lost more than 10 percent of their body weight. Consistent with past clinical research experience, Imcivree was generally well- resistant in both trials.Obesity caused by POMC, PCSK1, or LEPR gene defects is an ultra-rare disease caused by POMC, PCSK1, or LEPR gene variants that impair the MC4-subject path, the path path of the lower pasum that regulates hunger, energy consumption, and weight. People who are obese due to POMC, PCSK1, or LEPR gene defects face extreme, unsealable hunger from an early age, leading to severe obesity in early onset. As an MC4 subjector agitant, Imcivree aims to restore impaired MC4 subject pathact activity due to genetic defects upstream of the MC4 subject. Prior to Imcivree, there was no FDA-approved specific therapy to control obesity weight due to POMC, PCSK1, or LEPR defects. It is worth mentioning that in the first half of this year, researchers at Shanghai University of Science and Technology published a cooperative study on the "precise structure of MC4R in the human brain" published in the top academic journal Science, which debunked the mystery of MC4R and revealed how the subject binds to other molecules.Dr. David Meeker, President and CEO of Rhythm, said, "The approval of our first new drug is a significant milestone for the company, and we look forward to delivering on Imcivree's commitment to delivering on Imcivree's commitment to patients who are obese due to POMC, PCSK1, or LEPR defects, and to advancing a "first-in-class" precision therapy designed to directly address the root causes of obesity driven by genetic defects in the MC4 subject path. "With this approval, the FDA issued Rhythm with a Rare Pediatric Disease Priority Review Certificate (PRV).“ Many patients and families with these diseases face stigma associated with severe obesity. To manage the weight of this obese patient and control excessive food-seeking behavior, caregivers often lock cupboards and refrigerators and greatly limit their social activities," said Dr. Jennifer Miller of the University of Florida School of Medicine.Setmelanotide is a "first-in-class" black cortide-4-4-subject exciter. In addition to the approval of the new drug listing application (NDA) in the United States, setmelanotide's application for a market license (MAA) for the treatment of obese patients caused by POMC, PCSK1 or LEPR gene defects is currently under review by the European Medicines Agency (EMA). In addition, Rhythm is conducting a key Phase 3 trial in patients with Bardet-Biedl or Alström syndrome to assess the use of setmelanotide to reduce hunger and weight, with full data expected by the end of the fourth quarter of 2020 or the beginning of the first quarter of 2021.

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)References:1. Rhythm Pharmaceuticals Pharmaceuticals FDA Approval of IMCIVREE™ As First-Ever Therapy for Chronic Weight Management with Obesity to Due POMC, PCSK1 or LEPR Didy. Retrieved 2020-11-27, from