Epilepsy Society welcomes new medication for Dravet syndrome

The Epilepsy Society has welcomed a new experimental treatment for children with Dravet syndrome which researchers say is safe and can help reduce seizures dramatically.

Fergus Rugg Gunn, Consultant Neurologist & Honorary Associate Professor, Clinical Lead, Chalfont Centre for Epilepsy Society said: "Dravet syndrome affects one in every 15,000 babies and can have a devastating impact on their lives. This is an exciting treatment that targets the underlying genetic cause of the condition and offers real hope for those children and their families. More trials will be needed before the treatment can be made available for all children with Dravet syndrome, but these results are very promising."

The researchers have published the latest results of their initial trial and extension studies which have involved 81 children with Dravet syndrome in the UK and US.

The published data are from initial studies designed primarily to evaluate the safety and tolerability of Zorevunersen. The researchers also evaluated its effects on seizures, cognition, behavior and quality of life. A Phase Three study is currently underway to further evaluate the treatment.

Lead author Professor Helen Cross, Director and Professor of Childhood Epilepsy at the UCL Institute of Child Health and an Honorary Consultant in Paediatric Neurology at Great Ormond Street Hospital (GOSH), said: “I regularly see patients with hard-to-treat genetic epilepsies with impacts that go beyond seizures and it’s heart-breaking when treatment options are limited. This new treatment could help children with Dravet syndrome lead much healthier and happier lives.

"Overall, our findings showed that Zorevunersen is safe to use and well tolerated by most patients and supports further evaluation in the ongoing Phase Three study.”

Eighty-one children aged two to 18 took part in the initial trial. Patients in these studies had an average of 17 seizures per month before the trials started.

The 81 children were given up to 70mg of Zorevunersen by lumbar puncture, either as a single dose or with additional doses two or three months later over a six-month period.

Of those patients, 75 went on to take part in extension studies. Those patients continued to receive the drug every four months.

Patients given a 70mg dose in the initial phase of the trial saw their seizures reduce by between 59 per cent and 91 per cent over the first 20 months of the extension studies compared with the number of seizures they were having before the trial started.

Nineteen of the trial participants were patients at UK hospitals. As well as GOSH, those hospitals were Sheffield Children’s Hospital, Evelina London Children’s Hospital and The Royal Hospital for Children in Glasgow.

At GOSH, the trial was conducted at the National Institute of Health and Care Research’s Clinical Research Facility, which is a state-of-the-art facility dedicated to children taking part in experimental trials.

Dravet Syndrome UK Chair of Trustees Galia Wilson said: “We regularly see the devastating impact that this condition has on the lives of families. That’s why we’re so thrilled about these latest results from the initial zorevunersen clinical trials.

“We’re now looking forward to the Phase Three clinical trials taking place to see if the early promise we see here will translate into real hope for all those families currently affected by Dravet Syndrome.”