Intranasal carbetocin (LV-101; Levo Therapeutics), an investigational treatment for Prader-Willi syndrome (PWS), has been granted Fast Track designation by the Food and Drug Administration (FDA).

The intranasal formulation of carbetocin, an oxytocin analog, will be investigated in an  8-week placebo-controlled study (CARE-PWS) to assess the effectiveness, safety, and tolerability of the investigational product in patients with genetically-confirmed PWS. The primary outcome measures of the study will include change in hyperphagia as measured by the Hyperphagia Questionnaire for Clinical Trials total score, as well as change in obsessive and compulsive behaviors as measured by the Children’s Yale-Brown Obsessive-Compulsive Scale total score. The study will also include a long-term follow-up period (56 weeks) and an optional extension period after study Week 64 during which all patients will receive active treatment.

Results of a phase 2 trial evaluating intranasal carbetocin in PWS showed that the treatment was well-tolerated and significantly reduced hyperphagic symptoms when compared with placebo. In addition, reduction in compulsivity and an overall improvement in functioning was also noted following 14 days of treatment. As for safety, the incidence of adverse events was found to be similar between carbetocin and placebo.

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“We are thrilled to receive Fast Track designation from the FDA, which supports the understanding among the PWS community that this syndrome presents serious and life-threatening issues,” said Sara Cotter, CEO of Levo Therapeutics. “We look forward to finishing enrollment of this important clinical study in the coming months.”

Fast Track designation is granted to therapies for serious and life-threatening conditions so that an approved product can reach the market expeditiously.

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