Levo Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for LV-101 (intranasal carbetocin) for the treatment of Prader-Willi syndrome (PWS). A Phase 3 clinical study, CARE-PWS, of intranasal carbetocin for the treatment of PWS is currently underway and recruiting patients.
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.
CARE-PWS is a multi-center, randomized, double-blind, placebo-controlled study in approximately 175 patients ages 7-18 years old with Prader-Willi syndrome. The co-primary endpoints are change in hyperphagia (extreme hunger) as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Total Score versus placebo and change in obsessive and compulsive behaviors as measured by the Children’s Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) Total Score versus placebo. Both endpoints will be measured from baseline to Week 8. The study is currently underway at over 20 clinical study sites throughout the United States and Canada, with additional study sites anticipated to open in Australia soon.
Carbetocin is an analog of the naturally-occurring neuroendocrine hormone oxytocin. Carbetocin 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 potential benefit of intransal delivery of an oxytocin analog in patients with PWS may be gleaned from existing evidence of the specific neurologic actions of this hormone. Indeed, oxytocin is a potent anorexigenic hormone, and it is integral to the complex neural and gut networks associated with the homeostatic control of food intake, satiety, and energy balance.
About Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a complex, multisystem neurodevelopmental disorder that occurs in approximately 1 in 16,000 births. The underlying cause of PWS is the lack of expression of paternally-inherited imprinted genes on chromosome 15q11-q13. These genetic anomalies lead to a distinctive phenotype that includes mild to moderate levels of intellectual disability, compulsivity, growth hormone deficiency, life-threatening hyperphagia, and high risk of obesity.