Roche’s balovaptan obtains FDA breakthrough therapy designation

Roche has obtained breakthrough therapy designation from the US Food and Drug Administration (FDA) for its investigational drug balovaptan to treat patients with autism spectrum disorder (ASD).

Designed as a small molecule vasopressin 1a (V1a) receptor antagonist, balovaptan is reported to have demonstrated potential to improve social interaction and communication for people with ASD.

The FDA has granted the designation based on efficacy data obtained during the Phase II VANILLA trial of balovaptan in adults suffering from ASD.

According to the results, the drug candidate demonstrated favourable safety and tolerability profile.

It is expected that if these results are confirmed in further studies balovaptan can potentially be the first pharmacotherapy to aid core socialisation and communication symptoms of ASD.

share on: