Results from CARE-PWS Phase III study of intranasal carbetocin in the treatment of Prader-Willi syndrome released

Although the primary endpoint (evaluation of the 9.6mg dose) was not achieved, the 3.2mg dose was associated with a statistically significant improvement in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score (p=0.016; secondary endpoint, no further details given).

SPS commentary:

Carbetocin is an oxytocin analogue with improved receptor binding profile compared to oxytocin. An investigational intranasal form is currently in Phase III development for the treatment of hyperphagia and other linked symptoms (obsessive compulsive symptoms and anxiety) in patients with Prader-Willi syndrome, a complex genetic disorder.

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