FDA rejects New Drug Application for LV-101 (intranasal carbetocin) as a treatment for excessive overeating, anxiousness and distress associated with Prader-Willi syndrome (PWS)
FDA indicated that while the drug appeared to be safe and well-tolerated, efficacy data for the proposed 3.2-mg dose were not sufficient for approval. The agency recommended another clinical trial be conducted to confirm the data from the study on the 3.2-mg dose.
Source:
Biospace Inc.
SPS commentary:
Carbetocin is an analogue of the hormone oxytocin, engineered to have an improved receptor-binding profile compared to oxytocin, improving how it binds to the receptor while binding less to other vasopressin receptors. LV-101 is an intranasal form of the drug designed to be dosed in PWS patients three times a day with meals. In infancy, PWS is marked by weak muscle tone, problems eating, slow growth and developmental delay. Later, in early childhood, they develop an uncontrolled appetite, which leads to chronic overeating and obesity. It is also associated with mild to moderate cognitive and learning issues and behavioural problems.