M. Scott Perry, MD: Clinical Experience With Fenfluramine in Dravet Syndrome

April 26, 2020

The co-director of the Jane and John Justin Neurosciences Center at Cook Children’s Hospital discussed his experience with Zogenix’s investigational Dravet syndrome treatment in clinical trials and in the Expanded Access Program.

“This is a superior drug for Dravet syndrome. The seizure reductions I’m seeing, especially with convulsive seizures, in these kids is as significant as the phase 3 data.”

Despite the cancellation of the American Academy of Neurology (AAN) 2020 Annual Meeting data remains abound in the neurology space. One such dataset which was scheduled to be presented at AAN 2020 was that of the first 23 patients in the fenfluramine (Fintepla; Zogenix) Expanded Access Program (EAP), which was collected by M. Scott Perry, MD, medical director of neurology, and co-director, Jane and John Justin Neurosciences Center, Cook Children’s Hospital, and colleagues.

The antiseizure therapy is set for FDA review in June of this year, and these real-world data appear to confirm the reductions in seizure frequency that were observed in the pivotal clinical trials. Now, however, with further context of its use, NeurologyLive reached out to Perry to find out more about his experience with fenfluramine ahead of possible agency approval.

Perry highlighted some of the success that patients of his have had with the agent being used in combination with other therapies such as clobazam, valproate, stiripentol, and cannabidiol (CBD) through the EAP, as well as what other aspects of its use he would like to see explored in future assessments of the drug.

For more AAN 2020 coverage, click here.

REFERENCE

Perry MS, Wirrell E, Burkholder D, Galer BS, Gammaitoni A. Real-world Experience with ZX008 (Fenfluramine HCl) for the Treatment of Seizures in Dravet Syndrome: Initial Report from United States Expanded Access Program. Neurology. 2020;94(15 Suppl): 1985.