FDA rejects new drug application for internasal carbetocin to treat Prader-Willi syndrome
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The FDA’s Division of Psychiatry has rejected a new drug application for treating hyperphagia, anxiousness and distress associated with Prader-Willi syndrome, according to a press release.
As Healio previously reported, the FDA granted priority review for LV-101 (intranasal carbetocin, Levo Therapeutics) in July 2021. In a complete response letter, the FDA determined the therapy was safe and well tolerated, but efficacy for the proposed 3.2 mg dose was insufficient for approval.
The FDA is recommending an additional clinical study be conducted to confirm the results for the 3.2 mg dose. Levo is in discussions with the FDA regarding the design of the new study, according to the release.
“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,” Sara Cotter, CEO of Levo Therapeutics, said in the press release. “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.”
In top-line data from the CARE-PWS study published in August 2020, adults with Prader-Willi syndrome who received internasal carbetocin for 8 weeks had a significant reduction in hyperphagia with a 3.2 mg dose as evaluated by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score (P = .016) and in anxiety and distress behaviors, as evaluated by the Prader-Willi syndrome Anxiety and Distress Behaviors Questionnaire (P = .027). When the 3.2 mg dose arm and 9.6 mg dose arm of the study were pooled together, the change in HQ-CT score resulted in a P value of 0.55. There was no statistically significant effect for either a 3.2 mg dose or 9.6 mg dose on the Children’s Yale-Brown Obsessive Compulsive Scale.
“We remain steadfast in our support of carbetocin and all potential treatments for our community,” Theresa Strong, PhD, founding member and director of research programs at the Foundation for Prader-Willi Research, said in the press release. “We hope that, going forward, the FDA will recognize the tremendous unmet need and exercise the flexibility it has when considering new products for rare, serious diseases such as Prader-Willi syndrome.”
Caregivers who would like to be contacted as details on the new study become available can provide their information at www.pwsstudy.com.
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