Promising New Drug to Treat Rare Form of Epilepsy

Epilepsy treatment continues to advance and progress. We have recently discussed breakthroughs in minimally invasive surgery for reducing seizures, and now there’s even more good news.

A new drug has been called potentially life-changing for children with Dravet syndrome, a severe form of epilepsy.

In its early trials, the drug, called Zorevunersen, was shown to reduce seizures in children with Dravet syndrome by up to 91%.

What is Dravet Syndrome?

Dravet syndrome is a severe and rare genetic neurological condition that causes treatment-resistant epilepsy.

In most cases, Dravet syndrome is caused by a mutation in the gene SCN1A. This gene plays an important role in regulating electrical signals in the brain. When the gene is not working properly, the brain’s neurological activity becomes unbalanced, leading to seizures.

It is a lifelong condition that typically develops in infancy, usually first presenting itself in the form of a seizure when the child is between 3 and 12 months of age.

Generally, children with Dravet syndrome have normal development in the early stages, but as the seizures continue, they can lead to intellectual disability along with conditions such as autism and ADHD.

Dravet syndrome can cause a range of seizures, but the most common is a tonic-clonic seizure. This type of seizure affects both the brain and body, causing loss of consciousness and rapid muscle spasms, which can result in jerking of the limbs.

With Dravet Syndrome, seizures are usually very frequent, with some children experiencing hundreds of seizures a day. Even just a single tonic-clonic seizure can cause injury or be life-threatening to a child.

Early diagnosis and environmental changes can help to reduce the risks, but ultimately, living with Dravet syndrome severely impacts the lives of those affected by it. Now, though, there is a new medication providing great hope to patients with Dravet syndrome and their families.

Zorevunersen to Significantly Reduce Seizures

Zorevunersen is a promising new drug that does more than just treat symptoms; it targets the underlying cause of Dravet syndrome.

As mentioned earlier, Dravet syndrome is caused by a mutation in the gene SCN1A. SCN1A contributes to healthy brain activity by producing proteins known as sodium ion channels.

Generally, humans carry two healthy copies of this gene. In people with Dravet syndrome, though, one of the copies fails to produce the necessary amount of sodium ion proteins. Zorevunersen works to correct this issue by increasing the production of the sodium ion protein from the healthy copy of SCN1A.  

A study involving 81 patients with Dravet syndrome between the ages of 2 and 18 showed Zorevunerson capable of reducing the frequency of seizures by between 59% and 91%.

Besides monitoring the effectiveness of Zorevunerson in managing seizures, the study focused on the safety and tolerability of the drug. Results show it is generally well-tolerated with mild to moderate side effects. The most common side effect is post-lumbar puncture syndrome, as the drug is delivered directly into the cerebrospinal fluid (the fluid surrounding the brain and spinal cord) via a lumbar puncture or spinal tap.

Further studies involving larger groups of patients are already in process to truly determine the effectiveness of Zorevunerson. At the current pace, results from these larger trials are expected by mid-2027, which could initiate the New Drug Application (NDA) process sooner than expected.

While the results so far are compelling, we will need to wait for the completion of these larger trials before we can fully understand Zorevunersen’s impact on the epilepsy community.

Even so, there is real reason for optimism. What makes this drug especially noteworthy is that it not only treats symptoms; it targets the root cause of Dravet syndrome. In doing so, it has the potential to reduce seizures and contribute to improvements in cognitive and behavioural health.

This news is close to our hearts at Epilepsy Alarms UK, and we’re so pleased to be able to share this current information with you. We will follow the upcoming trials closely and strive to keep you updated on any important information regarding Zorevunerson. In the meantime, please see our blog for more of the latest news relating to epilepsy.