Special Episode: FDA Green-Lights Vamorolone for Duchenne Muscular Dystrophy

Barry J. Byrne, MD, PhD

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, "FDA Green-Lights Vamorolone for Duchenne Muscular Dystrophy," features an exclusive interview with Barry J.  Byrne, MD, PhD, chief medical advisor, Muscular Dystrophy Association, and associate chair of pediatrics and director of the Powell Gene Therapy Center at the University of Florida. He 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).^1-5

Click here to read more of NeurologyLive's coverage of the vamorolone approval.

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

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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