One of the most common types of mutations in the dystrophin gene occurs when a piece of the code in the middle of the gene is missing or deleted. By skipping additional segments of the dystrophin code called exons, the deletion can shift from an out-of-frame deletion to an in-frame deletion. Typically an in-frame deletion results in a smaller, but still functional, dystrophin protein. This shortened protein is expected to act in a similar way to normal dystrophin, and so relieve some symptoms of Duchenne and hopefully result in a more mild presentation. VYONDYS 53® is for use in eligible patients with a mutation in the dystrophin gene that is amenable to exon 53 skipping.

Status

VYONDYS 53 is indicated for the treatment of Duchenne in eligible patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping (FDA label document). This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

For additional resources regarding eligibility and access, view PPMD’s Insurance Access & Coverage Roadmap.

Sponsor

This program is sponsored by Sarepta Therapeutics.

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