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. Casimersen (SRP-4045) is designed to skip exon 45 in the dystrophin gene.
A Phase 3 study has reached targeted enrollment in the United States but is currently actively recruiting in other countries.
The FDA has accepted Sarepta’s New Drug Application (NDA) seeking accelerated approval for Casimersen, with a regulatory action date of February 25, 2021.
This program is sponsored by Sarepta Therapeutics.
OPEN LABEL EXTENSION
|An Extension Study to Evaluate Casimersen or Golodirsen|
Sarepta Presents at the PPMD 2019 Annual Conference
Sarepta Presents at the PPMD 2018 Annual Conference
Webinar: ESSENCE - Sarepta’s Exon 45 and 53 Skipping StudyPPMD and Sarepta Therapeutics hosted a webinar June 7, 2017 for an overview of Sarepta’s pivotal study investigating SRP-4045 and SRP-4053 for the treatment of Duchenne.