Special Episode: Trofinetide Approved for Rett Syndrome

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. For major FDA decisions in the field of neurology, we release 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 approval of trofinetide (Daybue; Acadia) in Rett syndrome. Offering commentary is Jeffrey L. Neul, MD, PhD, the Annette Schaffer Eskind Chair and director of the Vanderbilt Kennedy Center, and professor of pediatrics at Vanderbilt University Medical Center, and the lead investigator on the phase 3 study of the drug, called LAVENDER (NCT04181723). Trofinetide is a synthetic analog of the amino‐terminal tripeptide of IGF-1, and is now the first treatment approval for Rett syndrome, marking a milestone in the field of developmental disorder care. For more of NeurologyLive®'s coverage of trofinetide's approval, head here: FDA Approves Acadia's Trofinetide for Rett Syndrome Episode Breakdown: 0:30 – Trofinetide (Daybue; Acadia) approved for Rett syndrome 1:30 – Jeffrey L. Neul, MD, PhD, on the approval 4:10 – Prevalence of Rett syndrome and the impact on research 5:40 – Supporting data from the phase 3 LAVENDER study 7:20 – Safety data from the clinical development and managment of common adverse events 8:45 – Closing thoughts 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. Acadia Pharmaceuticals Announces U.S. FDA Approval of DAYBUE™ (trofinetide) for the Treatment of Rett Syndrome in Adult and Pediatric Patients Two Years of Age and Older News Release. Acadia Pharmaceuticals. March 10, 2023. Accessed March 13, 2023. https://acadia.com/media/news-releases/acadia-pharmaceuticals-announces-u-s-fda-approval-of-daybue-trofinetide-for-the-treatment-of-rett-syndrome-in-adult-and-pediatric-patients-two-years-of-age-and-older/ 2. Neul JL. Rett Syndrome. National Organization for Rare Diseases. Updated December 17, 2019. Accessed March 13, 2023. https://rarediseases.org/rare-diseases/rett-syndrome/