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


Mind Moments®, a podcast from NeurologyLive®, brings you exclusive interviews with Scott Demarest, MD, MSCS, and Kelly Knupp, MD, MSCS, FAES. [LISTEN TIME: 21 minutes]

Scott Demarest, MD, principal investigator for the Marigold trial, and neurologist and clinical director, Precision Medicine, Children’s Hospital Colorado

Scott Demarest, MD, MSCS

Kelly G. Knupp, MD, MSCS, FAES, associate professor of pediatrics and neurology, and codirector, neurology research, University of Colorado, Anschutz Campus, in Aurora, Colorado

Kelly G. Knupp, MD, MSCS, FAES

A special episode of the NeurologyLive® Mind Moments® podcast is now live! Scroll down to listen or click here to subscribe on your favorite streaming service.

The Mind Moments® podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis (MS), Parkinson disease, dementia, sleep disorders, and more.

This episode, "Ganaxolone Approved for CDD, Fenfluramine Approved for LGS," features exclusive interviews with 2 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, who was an investigator of the Marigold trial (NCT03572933) of ganaxolone (Ztalmy; Marinus Pharmaceuticals) in cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder, also known as CDD.1 The second is Kelly G. Knupp, MD, MSCS, FAES, pediatric neurologist, Children's Hospital Colorado, and associate professor of pediatrics-neurology, University of Colorado, who was an investigator of Study 1601 (NCT03355209) of fenfluramine (Fintepla; Zogenix/UCB) in Lennox-Gastaut syndrome (LGS).2

Click here to read more of NeurologyLive®'s coverage of the ganaxolone approval, and click here to read more of NeurologyLive®'s coverage of the fenfluramine approval.


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

Click here to subscribe to the Mind Moments® podcast. Be sure to leave a rating and review for the show. Thanks for listening!

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
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