M. Scott Perry, MD: Real-World Fenfluramine Data in Dravet Syndrome

April 25, 2020

The co-director of the Jane and John Justin Neurosciences Center at Cook Children’s Hospital discussed the real-world data collected from the Expanded Access Program for fenfluramine (Fintepla; Zogenix) in patients with Dravet syndrome.

“When we start the medicine, we keep it stable for 30 days before we start making changes in other meds, but we can make changes in other meds and I think that’s really the take-home [message].”

New real-world data on fenfluramine (Fintepla; Zogenix) that were accepted for presentation at the American Academy of Neurology (AAN) 2020 Annual Meeting suggest that similarly to the clinical trials, the therapy results in clinically meaningful responses in patients with Dravet syndrome.

Conducted by M. Scott Perry, MD, medical director of neurology, and co-director, Jane and John Justin Neurosciences Center, Cook Children’s Hospital, and colleagues, this initial report of the US Expanded Access Program for fenfluramine included 23 patients with Dravet syndrome with a mean age of 6.8 years, who had been treated for a median of 90 days. In total, 65% (n = 15) of the cohort reported meaningful global clinical improvement, defined as a rating of “very much improved” or “much improved.” The most common concomitant antiepileptic drugs (AEDs) were clobazam, valproate, stiripentol, and cannabidiol (CBD).

As fenfluramine is up for FDA review in June, these data mean quite a bit for those who are treating this patient population. In order to find out more about these data which were sent to be presented at AAN and what the clinical community needs to know, NeurologyLive caught up with Perry virtually to discuss.

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.