Experimental autism drug balovaptan granted Breakthrough Therapy Designation

The FDA has granted Breakthrough Therapy Designation for Roche’s (Basel, Switzerland) investigational autism drug, balovaptan. Balovaptan, an oral medication, is a vasopressin 1a (V1a) receptor antagonist intended for use in individuals with autism spectrum disorder (ASD). The V1a receptor has been implicated in the mediation of social behaviors that are typically challenging for individuals with ASD, in both animal and human trials. FDA Breakthrough Therapy Designation is intended to expedite the development and review of medications with early evidence of efficacy and potential benefit to patients. Currently no pharmacological agents exist to alleviate the social challenges experienced by sufferers of...