Roche’s Autism Drug Receives FDA Breakthrough Status

Roche announced on Monday that the U.S. Food and Drug Administration (FDA) has designated the company’s experimental autism drug balovaptan (RG7314) a breakthrough therapy.

Several studies suggest that balovaptan—a vasopressin 1a (V1a) receptor antagonist—may improve social interaction and communication in people with autism. There are currently no FDA-approved drugs for the core symptoms of autism such as problems with social interaction and engaging in repetitive behaviors.

According to a Roche statement, the FDA Breakthrough Therapy Designation for balovaptan “was primarily based on efficacy findings in the VANILLA (Vasopressin ANtagonist to Improve sociaL communication in Autism) study,” a phase 2 trial of the medication in adults with autism spectrum disorder (ASD). Balovaptan is currently in phase 2 trials in children and adolescents with ASD. ■