Kelly Knupp, MD: Fenfluramine in Lennox-Gastaut Syndrome

"One of the outcome majors that has worked really well are seizures associated with a drop. Kids with Dravet [syndrome] have a lot of types of seizures, but children with LGS really have a lot of types of seizures, including a lot of small seizures that can be hard to count.”

Full results of the phase 3 Study 1601 (NCT03355209) of the investigational agent fenfluramine (Fintepla; Zogenix) were recently presented virtually at the American Epilepsy Society (AES) Annual Meeting, December 4–8, 2020. The data showed that the trial met its primary end point, with the treatment demonstrating significant improvement in monthly drop in seizure frequency in patients with Lennox-Gastaut syndrome (LGS).

Speculation of whether fenfluramine may become the next FDA-approved treatment for patients with LGS has lingered for months after Zogenix originally released the topline data from the phase 3 trial in February 2020. Along with its robust efficacy, lead author Kelly Knupp, MD, pediatric neurologist and epilepsy specialist, Children’s Hospital Colorado, claims that no cases of valvular heart disease (VHD) or pulmonary arterial hypertension (PAH) were among 2 of the most notable findings within the study due to fenfluramine’s previous history with these issues.

Knupp sat down with NeurologyLive to discuss the findings of the study, including the most notable end points and the importance of this data in patients with LGS.

For more coverage of AES 2020, click here.

REFERENCE

Knupp K, Sullivan J, Nickels K, et al. Efficacy and safety of fintepla (fenfluramine) for the treatment of seizures associated with Lennox-Gastaut syndrome: a randomized, double-blind, placebo-controlled clinical trial. Presented at AES 2020 Annual Meeting; December 4–8, 2020. Abstract 852.