Rhythm, a biopharmaceutical company developing peptide therapeutics for rare genetic deficiencies that result in life-threatening metabolic disorders, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to setmelanotide, the company’s novel melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity. Setmelanotide is in Phase 2 clinical trials for the treatment of rare genetic disorders of obesity caused by MC4 pathway deficiencies.
“Patients with POMC deficiency obesity have extreme and unrelenting appetite and obesity because of impaired function in the MC4 pathway,” said Keith Gottesdiener, CEO of Rhythm. “We are pleased to receive this breakthrough designation and look forward to working closely with the FDA as we continue to advance the setmelanotide program.”
Breakthrough Therapy designation is granted by the FDA to expedite the development and review of therapeutics to treat serious or life-threatening conditions for which preliminary clinical evidence indicates that the therapy may demonstrate substantial improvement on at least one clinically significant endpoint over existing therapies. This designation conveys all FDA fast track program features, such as eligibility for rolling NDA submissions and priority review (if supported by clinical data at the time of NDA). Additionally, this designation provides more intensive involvement of FDA staff in a proactive, collaborative, cross-disciplinary review process. Setmelanotide is the first BTD to be awarded by FDA’s Division of Metabolism and Endocrinology Products, which is responsible for obesity and diabetes indications.
