FDA grants Breakthrough Therapy Designation for Roche’s balovaptan in autism spectrum disorder
OREANDA-NEWS. Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its investigational oral medicine balovaptan (previously known as RG7314), 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 behaviours that are challenging for individuals with ASD. FDA Breakthrough Therapy Designation is intended to expedite the development and review of medicines with early evidence of substantial potential clinical benefit to patients, or benefit patients without current treatment options.
“We are very pleased that the FDA has granted Breakthrough Therapy Designation for balovaptan, in recognition of its early promise for individuals with ASD,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “We look forward to working closely with the FDA in the hope that we can bring this medicine to these individuals as quickly as possible.”
ASD is a lifelong developmental condition that affects how an individual behaves, communicates and interacts with others.3 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.4 No pharmacological treatment currently exists to help improve the challenges that individuals may experience with social skills, repetitive behaviours, restrictive interests and communication. These core symptoms of ASD may be persistent and difficult to overcome.4
FDA Breakthrough Therapy Designation for balovaptan is primarily based on efficacy findings in the VANILLA (Vasopressin ANtagonist to Improve sociaL communication in Autism) study, a Phase II trial of balovaptan in adults with ASD.1 Trial results were presented at the International Congress for Autism Research (IMFAR) in May 2017, and also showed that balovaptan was safe and well tolerated by the subjects in the study.1 A Phase II trial (aV1ation) investigating balovaptan in children and adolescents with ASD is ongoing and other trials in ASD are being planned.5