The FDA Grants Breakthrough Therapy Designation for Balovaptan – Roche’s Product for Autism Spectrum Disorder (ASD)

Yes, the title is real and correct and the condition is ASD. The US Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation for Roche’s (RHHBY) drug balovaptan developed for ASD. Balovaptan has shown the potential to improve social interaction and communication in people with ASD.

Balovaptan is an investigational small molecule drug that acts as a vasopressin (V1a) receptor antagonist. Evidence from both human and animal studies implicate the V1a receptor in mediating and modulating key social behaviors that are challenging for individuals 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 specific drugs currently exist to help improve the challenges that individuals may experience with social skills, repetitive behaviors, restrictive interests, and communication.

If results from the VANILLA study are confirmed in further studies, articulating balovaptan potential to become the first pharmacotherapy to help improve core socialization and communication symptoms of ASD.

The 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.

FDA Breakthrough Therapy Designation for balovaptan is based primarily on efficacy findings in the VANILLA (Vasopressin ANtagonist to Improve sociaL communication in Autism) phase 2 study in adults with ASD.

The trial results were presented at the International Congress for Autism Research (IMFAR) in May 2017. In addition to the efficacy, the study also showed the product’s safety and tolerability by the subjects in the study.

For children and adolescents, a Phase 2 trial (aV1ation) investigating balovaptan is ongoing with other trials being planned. As mentioned above, ASD is associated with a wide range of symptoms, which can be grouped into two main categories: ‘core’ and ‘associated’.

Core symptoms are impairments of social interaction and communication, as well as repetitive behavior and restricted interests, all of which can significantly impact daily functioning.

Associated symptoms can include anxiety, seizures, language disability, sensory issues, attention deficits, mood alterations, sleep deficits, angry outburst, and self-injury.

ASD is approximately four times more commonly diagnosed in boys than in girls. The World Health Organization estimates that the global prevalence of ASD is approximately one in every 160 people and 0.3% of the global burden of disease. In the European Union, it is estimated that the prevalence of ASD can range from 57 to 67 per 10,000 children.

Prohost Observations

We hope that balovaptan will soon release the people with the autism spectrum disorder (ASD) from the chains that tie them to the world of isolation with no possible socialization, hence no escape from depression. It is no surprise that Roche Neuroscience is the creator and developer of balovaptan, which is already bringing hope to the autism spectrum disorder people. Roche Neuroscience develops treatments based on the biology of the nervous system. The products being developed or under development aim at improving the lives of people with chronic and potentially devastating neurological diseases.

This is fantastic news for the people with autism spectrum disorder. We love to see the product reach the people who badly in need of it to make them enjoy what they have missed during their isolation and depression.

We are confident that Roche Neuroscience will do whatever it takes to put balovaptan in the hands of the ASD people as soon as possible.

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