FDA Tells Levo It Won’t Approve Prader-Willi Syndrome Therapy without Additional Study

The U.S. Food and Drug Administration told Levo Therapeutics the data it submitted for LV-101, its experimental therapy to treat hyperphagia, anxiousness, and distress associated with Prader-Willi syndrome is insufficient to support approval and suggested the company conduct an additional study.

Photo: Sara Cotter, CEO of Levo Therapeutics.

Levo is currently in discussions with FDA regarding the design of this new study and continues to provide its drug to existing study patients.

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 associated hyperphagia (unrelenting hunger), to which a deficiency in oxytocin is believed to be contributory. LV-101 is a selective oxytocin-receptor agonist with more than 200 patient-years of safety data collected to date in individuals with PWS.

LV-101 is an intranasal form of 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 100 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 EU following vaginal birth, with an estimated cumulative exposure of more than 11 million patients. LV-101 is intended to be administered to patients with PWS three times each day with meals. It has been granted orphan drug and Fast Track designations from the FDA.

“We are disappointed by the outcome of FDA’s review of our application and the continued lack of treatments for the most significant symptoms of the syndrome,” said Sara Cotter, CEO of Levo Therapeutics. “We are hopeful that our discussions with FDA regarding the next study will be productive and that we can initiate enrollment of a confirmatory study later this year.”

Patient organizations expressed their support for approval of carbetocin.

“We hope that, going forward, FDA will recognize the tremendous unmet need and exercise the flexibility it has when considering new products for rare, serious diseases such as PWS,” said Theresa Strong, founding member and director of research programs at the Foundation for Prader-Willi Research.

Author: Rare Daily Staff