MedicalResearch.com Interview with:
Dr Kevin Sanders, MD
Principle Medical Director-Product Development Neuroscience
Assistant Professor, Departments of Psychiatry and Pediatrics
MedicalResearch.com: What is the background for this announcement?
Response: The FDA has granted Roche 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).
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. Trial results were first presented at the International Congress for Autism Research (IMFAR) in May 2017. Treatment effects were observed on the Vineland-II (secondary endpoint) and also demonstrated that balovaptan was safe and well tolerated by the subjects in the study. The Vineland-II is a scale that measures socialization, communication and daily living skills. This data was presented to the FDA and is part of the basis of the Breakthrough Designation.
MedicalResearch.com: What types of benefits may Balovaptan have on symptoms of ASD?
Response: We are hopeful that these suggestive results from the Phase II trial will be replicated in the ongoing Phase II pediatric trial (aV1ation) and planned Phase III trials.
Autism spectrum disorder 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 behaviours, restrictive interests and communication. These core symptoms of ASD may be persistent and difficult to overcome.
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 often challenging for individuals with ASD.
MedicalResearch.com: What should readers take away from your report?
Response: Significant unmet medical need exists in autism spectrum disorder, including limited treatment options for managing core symptoms. This Breakthrough Designation is the first of it’s kind for the field of Autism. We continue to move forward to replicate these exciting results across the age and gender range of ASD with hopes of bringing a new treatment option to patients and families with ASD.
MedicalResearch.com: What recommendations do you have for future research as a result of this report?
Response: A Phase II trial (aV1ation) investigating balovaptan in children and adolescents with ASD is ongoing and other trials in autism spectrum disorder are being planned.
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