Therapeutic Optimization of Stiripentol for Dravet Syndrome: James Wheless, MD, FAAP, FAAN, FAES

WATCH TIME: 4 minutes

"It's a great drug, but how do I make it easier for families to use, for the patients, and boost its efficacy just by getting it into the patient?"

A few years ago, the Dravet Syndrome Foundation (DSF), in collaboration with an international consensus panel, produced guidelines for the treatment of Dravet syndrome (DS), a rare, genetic disorder that causes a severe form of epilepsy that begins in infancy or early childhood. In the guidelines, the treatment recommendations note valproate as a first-line treatment, followed by fenfluramine, stiripentol, and clobazam as second-line agents. Cannabidiol and topiramate were considered third and fourth line therapies, respectively, while medications like carbamazepine, oxcarbazepine, lamotrigine, and phenytoin were listed as treatments to avoid.

At the 2024 American Epilepsy Society (AES) Annual Meeting, held December 6-10, in Los Angeles, California, a product theater session focused on the impact of DS, signs and symptoms of the disease, as well as the importance of early intervention. The presentation, given by James Wheless, MD, FAAP, FAAN, FAES, also highlighted the therapeutic utility of stiripentol, which has been one of the longest standing therapies to treat DS. Stiripentol, usually started at a dose of 5-10 mg/kg/day, is approved in combination with clobazam to treat seizures associated with DS in persons 6 months of age and older. In other countries, it is approved to be used in combination with both valproic acid and clobazam for seizures in DS.

During the meeting, Wheless, professor and chief of pediatric neurology and the Le Bonheur Chair in Pediatric Neurology at the University of Tennessee Health Science Center, gave an overview of stiripentol and its use. Despite being relatively new in the US, stiripentol has been used in Europe for over 30 years, providing extensive clinical experience and predictable outcomes. Wheless, an epilepsy expert, discussed the importance of proper administration, particularly taking the medication with food to avoid degradation in the stomach. He also suggested potential improvements in drug delivery, such as developing a ready-to-use liquid or extended-release formulation to simplfy dosing.

Click here for more AES 2024 coverage.