FDA grants breakthrough therapy designation to balovaptan for autism

The FDA granted breakthrough therapy designation to balovaptan in treating autism spectrum disorder, according to a press release from the therapy’s manufacturer.

Balovaptan (formerly known as RG7314, Roche), an investigational oral medication, is a vasopressin 1a receptor antagonist for patients with autism spectrum disorder (ASD). It has been shown to improve social interaction and communication in patients with ASD, according to the release.

The FDA based the designation on efficacy data from the phase 2 VANILLA trial of balovaptan in adults with ASD, with results showing that bolavaptan was safe and well tolerated. A phase 2 study of children and adolescents is ongoing, according to the release.

“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, MD, chief medical officer and head of global product development for Roche, said in the release. “We look forward to working closely with the FDA in the hope that we can bring this medicine to individuals as quickly as possible.”

ASD is much more commonly diagnosed in boys than girls, with an estimated 1 in 42 boys and 1 in 189 girls in the U.S. having ASD, according to the release.

At present, there is no pharmacological treatment to help improve “the challenges that individuals may experience with social skills, repetitive behaviors, restrictive interests and communication,” according the release.