The FDA has awarded a breakthrough therapy designation to the investigational oral drug balovaptan (previously known as RG7314, Roche), a vasopressin 1a (V1a) receptor antagonist for individuals with autism spectrum disorder (ASD). Balovaptan has shown the potential to improve social interaction and communication in people with ASD. Evidence from both human and animal studies implicate the V1a receptor in mediating and modulating key social behaviors that are challenging for patients with ASD.
ASD is a lifelong developmental condition that affects how an individual behaves, communicates, and interacts with others. It is referred to as a “spectrum disorder” because of the broad range and severity of symptoms that individuals with autism have, all of which can significantly impact their daily functioning. No pharmacological treatment currently exists to help improve the challenges that individuals may experience with social skills, repetitive behaviors, restrictive interests, and communication. These core symptoms of ASD may be persistent and difficult to overcome.
The FDA’s breakthrough therapy designation for balovaptan is primarily based on efficacy findings in the VANILLA study, a phase 2 trial of balovaptan in adults with ASD. Trial results showed that balovaptan was safe and well tolerated. A phase 2 trial (aV1ation) investigating balovaptan in children and adolescents with ASD is ongoing and other trials in ASD are being planned. If results from the VANILLA study are confirmed in further studies, balovaptan has the potential to be the first pharmacotherapy to help improve core socialization and communication symptoms of ASD, according to Roche.
Source: PipelineReview.com; January 29, 2018.