Panel Votes Down Prader-Willi Syndrome Drug

FDA’s Psychopharmacologic Drugs Advisory Committee 11/5 voted 12 to 1 that a Levo Therapeutics NDA for carbetocin nasal spray and its use for treating Prader-Willi syndrome (PWS) did not show significant evidence of effectiveness. The submission is seeking approval for the drug to treat PWS-related hyperphagia, anxiety, and distress. “The primary endpoints were definitively negative; secondary endpoints were encouraging but questionable, and not even statistically significant when appropriately analyzed,” one panel member said.

Carbetocin is an analog of the naturally-occurring neuroendocrine hormone oxytocin. It was “designed to have an improved receptor binding profile compared to oxytocin, with greater affinity for the oxytocin receptor and lower affinity for related vasopressin receptors,” the company says. It is approved in over 90 countries outside the U.S. for preventing uterine atony and excessive bleeding during cesarean section delivery.