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.

Episode Breakdown:

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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/