Pharma Focus Europe

Roche’s Balovaptan Gets FDA’s Breakthrough Therapy Designation

Roche announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its investigational oral medicine Balovaptan.

It is a vasopressin 1a (V1a) receptor antagonist for individuals with Autism Spectrum Disorder (ASD).

The V1a receptor has a role in mediating and modulating key social behaviours associated with individuals with the condition.

Balovaptan has shown the potential to improve social interaction and communication in people with ASD.

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

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.

Core symptoms of ASD are impairments of social interaction and communication, as well as repetitive behaviour 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.

Furthermore, ASD is a lifelong developmental condition that affects how an individual behaves, communicates, and interacts with others. An individual with autism faces various symptoms, which they significantly impact their daily functioning.

FDA has granted Breakthrough Therapy Designation for Balovaptan 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.

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