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Roche’s autism drug scores FDA breakthrough status

Balovaptan could become the first drug to treat autism spectrum disorder


A drug that may improve social interaction and communication in people with autism spectrum disorder (ASD) has been granted Breakthrough Therapy Designation by the US Food and Drug Administration.

Roche’s oral investigational medicine Balovaptan could become the first drug approved for the disorder, and if it does it would mark a very big win for the Swiss pharma giant.

Sandra Horning, chief medical officer and head of global product development for Roche, said: “We are very pleased that the FDA has granted Breakthrough Therapy Designation for balovaptan, in recognition of its early promise for individuals with ASD.”

Horning added: “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.”

The FDA’s designation bucks the regulator’s usual trend of awarding Breakthrough Therapy Designations to oncology candidates.

Roche’s neuroscience drug met the FDA requirements mostly based on efficacy findings from the VANILLA study, a phase II trial of balovaptan in adults with ASD.

Reported at the International Congress for Autism Research in May last year, data from the trial demonstrated that the drug was ‘safe’ and ‘well tolerated’ by participants involved in the study.

Another phase II trial investigating the drug is currently ongoing, but for children and adolescents with ASD.

ASD is categorised as a lifelong developmental condition that affects how an individual behaves, communicates and interacts with others. The World Health Organization (WHO) estimates that the global prevalence of ASD is approximately one in every 160 people.

Article by
Gemma Jones

1st February 2018

From: Regulatory



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