JAN 2022 – A New Drug Application for LV-101 (intranasal carbetocin) was granted FDA Priority Review last year for the treatment of hyperphagia and behavioural symptoms in PWS, but ultimately, the review was unsuccessful as there were concerns about efficacy.
Following the positive results observed on hyperphagia, anxiety and distress symptoms at the lower 3.2mg dose in the phase 3 trial (CARE-PWS) and during the extension study, the negative vote by the FDA advisory committee in November last year surprised many in the PWS community. It was a disappointing outcome, especially considering the combined efforts of Levo Therapeutics, families, FPWR and PWSA(USA) to support the application, which included compelling testimonies given by families involved in the trial at the FDA virtual review meeting. There is much excitement about carbetocin because it is the closest oxytocin-like therapeutic option to being approved for use in PWS.
In January 2022, the FDA explained in a full response letter that, while LV-101 appears to be generally safe and well tolerated, the efficacy data available for the proposed 3.2 mg dose were insufficient and they recommended an additional clinical study be conducted to confirm results for this dose. Levo Therapeutics is currently in discussions with the FDA regarding the design of this new study and continues to provide carbetocin to existing study patients.