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FDA fast tracks intranasal oxytocin analogue for Prader-Willi syndrome
The FDA this week granted fast track designation for intranasal carbetocin, an oxytocin analogue, for the treatment of Prader-Willi syndrome, according to a press release from Levo Therapeutics.
Carbetocin was designed to have an improved receptor binding profile when compared with oxytocin, with greater affinity for the oxytocin receptor and lower affinity for related vasopressin receptors, according to Levo. The drug is approved in more than 90 countries outside the United States for the prevention of uterine atony and excessive bleeding during cesarean section delivery, with an estimated cumulative exposure of more than 10 million patients. LV-101 is an investigational intranasal form of carbetocin, intended to be administered to people with Prader-Willi syndrome three times daily before meals.
“We are thrilled to receive fast track designation from the FDA, which supports the understanding among the Prader-Willi syndrome community that this syndrome presents serious and life-threatening issues,” Sara Cotter, CEO of Levo Therapeutics, said in the release. “Levo is committed to developing impactful treatments for patients with Prader-Willi syndrome, and our phase 3 study is designed to see whether intranasal carbetocin provides one such treatment. We look forward to finishing enrollment of this important clinical study in the coming months.”
The FDA may designate a drug as a fast track product if the drug is intended, whether alone or in combination with one or more other drugs, for the treatment of a serious or life-threatening disease or condition, and it demonstrates the potential to address unmet medical needs for the disease or condition. Fast track designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously.
The phase 3 CARE-PWS study, a randomized, double-blind, placebo-controlled trial, is enrolling study participants at more than 20 clinical study sites throughout the U.S. and Canada, and Levo anticipates additional study sites opening in Australia soon. Researchers will assess the effectiveness, safety and tolerability of LV-101 for participants with Prader-Willi syndrome. Effectiveness will be measured using both caregiver-reported and clinician-reported measures of hyperphagia, obsessive and compulsive behaviors, and anxiety. Safety and tolerability will be measured by adverse events, laboratory tests and physical exams.
All participants will receive active treatment with LV-101 after the 8-week placebo-controlled period, during a long-term follow-up period of 56 weeks. At week 8, participants assigned placebo will be assigned one of two LV-101 doses, administered three times per day before meals. The study also includes an optional extension period, where participants in the long-term follow-up period will have an opportunity to continue receiving LV-101. – by Regina Schaffer
Disclosure: Cotter is CEO of Levo Therapeutics.