Clinical TopicsMusculoskeletal/OrthopedicsNewsPharmacologyRare Conditions

Potential new targeted therapy for Duchenne muscular dystrophy

By: Lydia L. Kim, Digital Content Editor

The U.S. Food and Drug Administration (FDA) recently granted accelerated approval for Sarepta Therapeutics’ golodirsen (Vyondys 53), an injection for patients with Duchenne muscular dystrophy (DMD) with a mutation in the dystrophin gene “that is amenable to exon 53 skipping.”

DMD is a rare genetic disorder that causes progressive muscle deterioration and weakness. Symptoms of DMD usually are first observed during childhood (between 3 and 5 years of age), and will progress over time, frequently causing death when patients reach their 20s or 30s. The condition is more common in males than females, with a diagnostic rate of one in every 3,600 male infants worldwide.

The director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research describes the impact of golodirsen: “The FDA recognizes the urgent need for new medical treatments for serious neurological disorders and we have a long-standing commitment to working with researchers, drug companies, and patients to facilitate the development and approval of treatments for rare diseases. With today’s accelerated approval, patients with Duchenne—a rare and devastating disease—who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping will now have available the first treatment targeted specifically for this disease subtype… Use of the accelerated approval pathway will make Vyondys 53 available to patients based on initial data and we look forward to learning more about the drug’s clinical benefit from the ongoing confirmatory clinical trial.”

Golodirsen previously was granted fast track, priority review, and orphan drug designations from the FDA and is an exciting development for the DMD community.

Please read more about the approval here.


The views and opinions expressed here are those of the author and do not necessarily reflect the opinions or recommendations of the American Nurses Association, the Editorial Advisory Board members, or the Publisher, Editors and staff of American Nurse Journal. This has not been peer reviewed.

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