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Roche Gets Breakthrough Status for Experimental Autism Drug

The designation could speed the development of the drug, with Roche expecting to submit for approval in 2020.

Roche Gets Breakthrough Status for Experimental Autism Drug

By: Sarah Hand, M.Sc.

Posted on: in News | Biotech News

According to a report by Reuters, Roche has been given Breakthrough Status designation from the FDA for a drug which is designed to treat autism spectrum disorders. The designation could speed the development of the drug, with Roche expecting to submit for approval in 2020.

Balovaptan blocks the V1a vasopressin receptor subtype, which could modulate the activity of the vasopressin hormone. Vasopressin improves inter-neuronal communication in the brain, and may also help support social interaction.

Individuals with autism with higher levels of the neuropeptide in the blood show better social functioning, suggesting that modulation of vasopressin could help treat some of the symptoms of the condition. Roche’s balovaptan is currently in Phase II of clinical development.

“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,” said Roche Chief Medical Officer Sandra Horning.

Stanford scientists are concurrently testing the effects of vasopressin itself on individuals with autism. In a small study of 50 patients, the researchers found that administering vasopressin via a nasal spray was associated with improvements in social functioning. These promising results have prompted the Stanford team to test the drug in a study of 100 autistic patients.

However, some have expressed concerns that this systemic supplementation of the neuropeptide could have negative side effects, including increasing aggressive behaviour. Roche’s drug aims to block the activity of vasopressin at one receptor to allow it to have an effect on social functioning without increasing the overall concentration of the hormone.

Roche’s preliminary results come from a study of adult males with autism. While the results failed to show a statistically significant improvement in social functioning, higher doses were associated with a significant improvement on an adaptive behavior rating scale.


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