Special Episode: FDA Greenlights Vamorolone for Duchenne Muscular Dystrophy

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 vamorolone (Agamree; Santhera Pharmaceuticals) oral suspension 40 mg/mL for the treatment of patients with Duchenne muscular dystrophy (DMD) aged 2 years and older. The therapy is set to be acquired by Catalyst Pharmaceuticals soon, and is a first-in-class dissociative steroid that aims to retain the anti-inflammatory activity of corticosteroids while decreasing the deleterious adverse events (AEs). We spoke with Barry J. Byrne, MD, PhD, professor and associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida, who discussed the recent FDA approval of Santhera Pharmaceuticals investigational agent vamorolone (now marketed as Agamree, and soon to be acquired by Catalyst Pharmaceuticals in North America), its potential to affect the treatment paradigm as a first-in-class dissociative steroid, and important efficacy and safety information from the phase 2b VISION-DMD study (NCT03439670). For more of NeurologyLive's coverage of vamorolone's approval, head here: FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy Episode Breakdown: 0:30 – Vamorolone approved for Duchenne muscular dystrophy 2:20 – Barry J. Byrne, MD, PhD, on his immediate reaction 3:25 – Safety data on vamorolone 4:00 – Byrne on the importance of bone mineralization in DMD 5:10 – Phase 2b efficacy data from VISION-DMD 5:45 – Byrne with context on the efficacy data 7:05 – Byrne with context on the landscape of DMD therapeutic development and personalization approaches 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. Catalyst Pharmaceuticals Reports FDA Approval of AGAMREE® (vamorolone) for Duchenne Muscular Dystrophy Granted to Santhera Pharmaceuticals. News release. Catalyst Pharmaceuticals. October 26, 2023. Accessed October 26, 2023. https://www.globenewswire.com/en/news-release/2023/10/26/2767947/13009/en/Catalyst-Pharmaceuticals-Reports-FDA-Approval-of-AGAMREE-vamorolone-for-Duchenne-Muscular-Dystrophy-Granted-to-Santhera-Pharmaceuticals.html 2. Santhera and ReveraGen announce positive and statistically highly significant topline results with vamorolone in pivotal VISION-DMD study. News release. Santhera. June 1, 2021. Accessed February 1, 2023. https://www.globenewswire.com/news-release/2021/06/01/2239124/0/en/Santhera-and-ReveraGen-Announce-Positive-and-Statistically-Highly-Significant-Topline-Results-with-Vamorolone-in-Pivotal-VISION-DMD-Study.html 3. Dang U, Guglieri M, Clemens PR, et al. Delayed start analysis of efficacy outcomes in placebo to vamorolone crossover participants in VBP15-004. Presented at MDA Clinical and Scientific Conference; March 13-16. Poster 89. 4. Muscular Dystrophy Association-Supported Drug AGAMREE® (vamorolone) Approved for the Treatment of Duchenne Muscular Dystrophy by the FDA. News release. MDA. October 26, 2023. Accessed October 26, 2023. https://www.mda.org/press-releases/mda-supported-drug-agamree-vamorolone-approved-for-the-treatment-of-duchenne-muscular-dystrophy-by-the-fda 5. Guglieri M, Clemens PR, Perlman SJ, et al. Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: a randomized clinical trial. JAMA Neurol. Published online August 29, 2022. doi:10.1001/jamaneurol.2022.2480