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 givinostat (Duvyzat; Italfarmaco) for the treatment of Duchenne muscular dystrophy (DMD). The therapy, a proprietary histone deacetylase (HDAC) inhibitor, was approved as the first nonsteroidal drug for patients with all genetic variants of DMD. The supporting data for the approval of givinostat comes from the phase 3 EPIDYS trial (NCT02851797). EPIDYS, a randomized, double-blind, placebo-controlled, multicenter study, included 179 ambulant male individuals who were randomly assigned 2:1 to either oral givinostat or placebo for an 18-month treatment period. Following the approval, Sharon Hesterlee, PhD, executive vice president and chief research officer of the Muscular Dystrophy Association, sat down to discuss the significance of the approval and how it changes the care for patients with DMD. She spoke specifically about the mechanism of action of the therapy, 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. 

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 

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