Joseph E. Sullivan, MD, Professor of Neurology & Pediatrics at University of California San Francisco and Director of Benioff Children’s Hospital Pediatric Epilepsy Center of Excellence, discusses zovrevunersen for the treatment of patients with Dravet syndrome.

 

Dravet syndrome is the most severe of a group of conditions known as SCN1A-related seizure disorders. Symptoms include seizures which first occur in infancy that are often triggered by high temperatures (febrile seizures). In childhood, many types of seizures may occur and they may increase in frequency and be difficult to treat. Other symptoms include loss of motor skills, intellectual disability, speech impairment, and difficulty with movement. Most cases of Dravet syndrome occur when the SCN1A gene is not working correctly.

Data was presented at the American Epilepsy Society (AES) 2024 Annual Meeting regarding zovrevunersen, an investigational antisense oligonucleotide for the treatment of Dravet syndrome. It is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant copy of SCN1A, reducing the occurrence of seizures and associated symptoms. Zorevunersen has been granted orphan drug designation, rare pediatric disease designation, and breakthrough therapy designation by the U.S. Food and Drug Administration (FDA).

In the phase 1/2a open-label extension study of zovrevunersen, nine patients were treated with an initial two or three 70mg doses, folllowed by a 45mg maintenance dose. When treated with this medication in addition to anti-seizure medications, sustainable and durable reductions in seizure frequency were observed. Improvements were also noted in multiple measures of cognition and behavior with ongoing treatment through two years. Tolerability of the treatment has also been favorable.

This data supports the proposed phase 3 study regimen of zovrevunersen for Dravet syndrome.

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To learn more about rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/