FDA Calls Roche's Autism Drug a Breakthrough

An experimental Roche drug designed to improve social interaction in people with autism scored the U.S. Food and Drug Administration’s Breakthrough Therapy Designation.

In an announcement this morning, Roche said the FDA awarded the designation to its experimental autism treatment balovaptan (RG7314). A vasopressin 1a (V1a) receptor antagonist, balovaptan has shown the potential to improve social interaction and communication in people with autism spectrum disorder. Roche said the drug’s efficacy has supporting evidence from both human and animal studies that implicate the V1a receptor in mediating and modulating key social behaviors in people with ASD.

The FDA’s 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 II trial of balovaptan in adults with ASD. A Phase II trial (aV1ation) investigating balovaptan in children and adolescents with ASD is ongoing and other trials in ASD are being planned, Roche said.  

The 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. Each of the symptoms can significantly impact the daily functioning of an ASD patient’s life. Currently, there are no approved treatments for ASD that are designed to "help improve the challenges that individuals may experience with social skills, repetitive behaviors, restrictive interests and communication," Roche said. These core symptoms of ASD may be persistent and difficult to overcome, the company added.

"We are very pleased that the FDA has granted Breakthrough Therapy Designation for balovaptan, in recognition of its early promise for individuals with ASD," Sandra Horning, Roche’s chief medical officer and head of global product development, said in a statement. "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 affects boys more often than girls. In the United States, it is estimated that one in 42 boys and one in 189 girls have ASD, Roche said.

With the Breakthrough Therapy Designation in hand, Roche may attempt to speed up the approval process for balovaptan. Writing in Endpoints News, John Carroll noted that Roche had been looking at a potential filing for regulatory approval by 2020, but could attempt to push that up. In the same breath though, Carroll noted that Roche’s pRED R&D unit, which has been working on balovaptan, is not known for "rapid development programs."