Special Episode: FDA Approves HDAC Inhibitor Givinostat for Duchenne Muscular Dystrophy

Sharon Hesterlee, 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 Approves HDAC Inhibitor Givinostat for Duchenne Muscular Dystrophy," features an exclusive interview with Sharon Hesterlee, PhD, executive vice president and chief research officer of the Muscular Dystrophy Association, who sat down to discuss the significance of the FDA approval of givinostat (Duvyzat; Italfarmaco) and how it changes the care for patients with DMD. She spoke specifically about the mechanism of action of the therapy as a proprietary histone deacetylase (HDAC) inhibitor, its safety profile, and how it may be used with other agents. In addition, she discussed other related topics on gene therapy and unmet needs for this patient population.^1,2

For more of NeurologyLive's coverage of givinostat's approval, head here: FDA Approves Italfarmaco's Givinostat for Duchenne Muscular Dystrophy

EPISODE BREAKDOWN

• 0:30 – Givinostat approved for Duchenne muscular dystrophy
• 2:00 – Sharon Hesterlee, PhD, on the approval's implications
• 3:35 – Positive downstream effects of the approval
• 4:50 – Hersterlee on the safety profile of givinostat
• 5:40 – Promising outlook of the DMD field
• 6:45 – Overcoming roadblocks involved with gene therapy 
• 8:55 – Remaining unmet needs for patients with Duchenne
• 10:15 – Closing thoughts on the approval 

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

REFERENCES
1. FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy. FDA. News release. March 21, 2024. Accessed March 29, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy
2. Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024;23(4):393-403. doi:10.1016/S1474-4422(24)00036-X