Special Episode: Ganaxolone Approved for CDD, Fenfluramine Approved for LGS

Welcome to this special episode of the NeurologyLive® Mind Moments™ podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In 2022 and onward, whenever the FDA makes a major decision for the field of neurology, we'll be releasing short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question. In this episode, we're covering the recent approvals of ganaxolone (Ztalmy; Marinus Pharmaceuticals) in cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder, also known as CDD, as well as fenfluramine (Fintepla; Zogenix/UCB) in Lennox-Gastaut syndrome (LGS). Offering commentary is a pair of experts. The first is Scott Demarest, MD, MSCS, neurologist and clinical director of precision medicine, Children’s Hospital Colorado, and assistant professor of pediatrics-neurology, University of Colorado, and an investigator of the Marigold trial (NCT03572933). The second is Kelly G. Knupp, MD, MSCS, FAES, pediatric neurologist, Children's Hospital Colorado, and associate professor of pediatrics-neurology, University of Colorado, and an investigator of Study 1601 (NCT03355209). For more of NeurologyLive®'s coverage of ganaxolone's approval, head here: Ganaxolone Wins Landmark Approval for CDKL5 Deficiency Disorder. For more of NeurologyLive®'s coverage of fenfluramine's approval, head here: Fenfluramine FDA-Approved for Treatment of Lennox-Gastaut Syndrome. For more coverage of epilepsy and seizure disorders, head to NeurologyLive®'s Epilepsy clinical focus page. Episode Breakdown: 0:30 – Ganaxolone (Ztalmy; Marinus) approved for CDD 2:30 – Scott Demarest, MD, with his reaction to the approval 3:00 – Data from the Marigold study of ganaxolone 4:45 – Safety profile of ganaxolone in development 5:10 – Demarest on the landscape of care for CDD 6:20 – Clinical takeaways ahead of DEA scheduling for ganaxolone 8:40 – Fenfluramine (Fintepla; Zogenix/UCB) approved for LGS 10:55 – Kelly Knupp, MD, with her reaction to the new indication 11:30 – Efficacy data on fenfluramine in LGS 13:50 – Safety profile in LGS and Dravet syndrome 16:05 – Knupp on the potential to improve quality of life and optimal use Thanks for listening to the NeurologyLive® Mind Moments™ podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com. REFERENCES 1. Marinus Pharmaceuticals Announces FDA Approval of ZTALMY® (ganaxolone) for CDKL5 Deficiency Disorder. News release. March 18, 2022. Accessed March 21, 2022. https://www.businesswire.com/news/home/20220318005282/en/Marinus-Pharmaceuticals-Announces-FDA-Approval-of-ZTALMY%C2%AE-ganaxolone-for-CDKL5-Deficiency-Disorder 2. FINTEPLA® (fenfluramine) Oral Solution Now FDA Approved for Treatment of Seizures Associated with Lennox-Gastaut Syndrome (LGS). News release. UCB. March 28, 2022. Accessed March 28, 2022. https://finance.yahoo.com/news/fintepla-fenfluramine-oral-solution-now-050000058.html