Rare Daily Staff

The U.S. Food and Drug Administration granted Levo Therapeutics Priority Review for its application for approval for its experimental therapy LV-101 as a treatment for hyperphagia and behavioral distress associated with Prader-Willi syndrome.

The FDA grants a six-month Priority Review to applications for drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness. Levo expects the FDA’s decision on the approval of LV-101 by the end of this year.

Prader-Willi syndrome (PWS) is a rare, complex, neurodevelopmental disorder that occurs in approximately 1 in 16,000 births and is characterized by a false state of starvation and unrelenting hunger, to which a deficiency in oxytocin is believed to be contributory. LV-101 is a selective oxytocin-receptor agonist.

LV-101 is a form of intranasal-delivered carbetocin, 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. Carbetocin is approved in more than 90 countries outside the United States for the prevention of uterine atony and excessive bleeding during cesarean section delivery, as well as newly approved in the European Union following vaginal birth, with an estimated cumulative exposure of over 10 million patients. The FDA previously granted LV-101 Orphan Drug and Fast Track designations.

“If approved, intranasal carbetocin will be the first specific treatment for the significant and disabling behavioral symptoms of PWS,” said Sara Cotter, CEO of Levo Therapeutics.

Photo: Sara Cotter, CEO of Levo Therapeutics.

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