FDA Approved Breakthrough Therapy Designation to Stoke Therapeutics' Zorevunersen for Dravet Syndrome
Stoke Therapeutics has announced that its investigational drug, zorevunersen, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA). This designation is for Zorevunersen’s potential use in treating Dravet syndrome with a confirmed mutation in the SCN1A gene that is not associated with gain-of-function.
Zorevunersen is being developed as the first disease-modifying therapy for Dravet syndrome, a severe form of epilepsy.
The Breakthrough Therapy Designation follows encouraging results from clinical trials showing that zorevunersen significantly reduces seizure frequency and improves cognitive and behavioural outcomes in patients.
These benefits were seen alongside the current standard anti-seizure medications. The drug was well tolerated in the studies, with over 600 doses administered to patients, some of whom have been treated for over three years.
Dravet syndrome is a severe genetic condition that causes frequent, prolonged seizures, beginning in infancy. It is difficult to treat and can lead to intellectual disabilities, developmental delays, and other serious health issues. People with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy (SUDEP). There are currently no disease-modifying treatments for the condition.
Zorevunersen works by restoring normal levels of the NaV1.1 protein, which is essential for proper brain function. The drug is designed to address the root cause of Dravet syndrome by using the non-mutant copy of the SCN1A gene to increase protein expression. This approach aims not only to reduce seizures but also to address other non-seizure-related health problems associated with the disease.
Zorevunersen has received orphan drug designation from both the FDA and the European Medicines Agency (EMA) and is recognised by the FDA as a potential treatment for a rare pediatric disease.
