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Sarepta Therapeutics to seek US approval for second DMD therapy

Sarepta Therapeutics Inc. said it will seek U.S. Food and Drug Administration approval for a second drug to treat Duchenne muscular dystrophy, a genetic disorder which primarily affects boys.

The company said it plans to file an application for SRP-4053, which is also known as golodirsen, by the end of the year.

According to the Boston Business Journal, golodirsen works by skipping over a genetic mutation that causes the muscle-wasting disease. Specifically, the drug skips over a mutation in a DNA sequence called exon 53 which affects about 8% of Duchenne patients.

The company's earlier drug, Exondys 51, works by skipping a different sequence: exon 51, which affects about 13% of boys diagnosed with Duchenne.

The Cambridge, Mass.-based biopharmaceutical company said it will seek accelerated approval for the medicine.