FDA grants priority review for intranasal Prader-Willi syndrome therapy
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The FDA granted priority review for a new drug application for an intranasal oxytocin analogue to reduce hyperphagia and behavioral distress associated with Prader-Willi syndrome, according to an industry press release.
A 6-month priority review will accelerate the review process and a decision from the FDA regarding the approval of LV-101 (intranasal carbetocin, Levo Therapeutics) should be made by the end of the year, according to the release.
“We are genuinely appreciative that FDA has accepted our NDA for priority review,” Sara Cotter, CEO of Levo Therapeutics, said in the release. “We are looking forward to working with the [FDA] during the coming months. If approved, intranasal carbetocin will be the first specific treatment for the significant and disabling behavioral symptoms of Prader-Willi syndrome.”
As Healio previously reported, top-line results from the CARE-PWS study, reported in August 2020, showed that adults with Prader-Willi syndrome who received intranasal oxytocin for 8 weeks reported a significant reduction in hyperphagia and symptoms of anxiety and distress.
Levo stated that statistical significance was achieved with the 3.2 mg dose as evaluated by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score (P = .016). When pooling the two dose arms of LV-101, per a prespecified analysis, the change in HQ-CT score from baseline to week 8 resulted in a P value of .055. Consistency in benefit/response was observed in the 3.2 mg dose arm across other key secondary endpoints, including clinical global impression of change (P = .027) and anxiety and distress behaviors, as evaluated by the Prader-Willi syndrome Anxiety and Distress Behaviors Questionnaire (P = .027). Neither dose demonstrated a statistically significant effect on the Children’s Yale-Brown Obsessive Compulsive Scale.
“Currently there are no therapies approved by FDA to treat the most challenging aspects of Prader-Willi syndrome, namely the constant hunger and distress that substantially impact patients and families,” Paige Rivard, CEO of the Prader-Willi Syndrome Association (PWSA) USA, said in the release. “PWSA USA has been advocating for patients and families since 1975, and we are incredibly excited that, for the first time, a treatment has made it this far in the development process.”
“This application is a substantial milestone for the PWS community, building on extensive scientific investigation of the neurobiological underpinnings of the syndrome,” Theresa Strong, PhD, founding member and director of research programs at the Foundation for Prader-Willi Research, said in the release. “We look forward to continuing to help the PWS community share its perspective on the challenges that our loved ones face, our treatment preferences, and how LV-101 presents an opportunity to address these needs.”
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