The director of the Pediatric Epilepsy Center at UCSF Benioff Children’s Hospital discussed the findings and the importance of discovering more about mechanisms of action.
“A lot of the focus is obviously on seizure reduction because that’s what gets you regulatory approval, but we’re looking at a lot of other secondary measures—the non-seizure related outcomes.”
Fenfluramine HCI oral solution, otherwise known as ZX008, is currently being investigated for the adjunctive treatment of seizures associated with Dravet syndrome. Data from Study, 1, a phase 3 clinical trial of the therapy, was recently reported at the American Epilepsy Society’s annual meeting in New Orleans, Louisiana, which showed a reduction in seizure frequency.
Additionally, an open-label extension study was conducted which showed these benefits extend over the long-term. For Joseph Sullivan, MD, the director of the Pediatric Epilepsy Center at UCSF Benioff Children’s Hospital, these results are incredibly positive, but a “pleasant surprise” for him was the additional finding that some patients experienced an improvement in executive function.
Although this may be attributable to the reduction in seizures, Sullivan posited that it could also be due to fenfluramine’s mechanism of action. To discuss the findings and the importance of discovering more about mechanisms of action, he sat with NeurologyLive in an interview.