Pharmaceutical

Sarepta Therapeutics (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced today that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat those patients with Duchenne muscular dystrophy who have genetic mutations subject to skipping exon 53 …

Sarepta Therapeutics (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced today that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat those patients with Duchenne muscular dystrophy who have genetic mutations subject to skipping exon 53 of the Duchenne gene.

As quoted in the press release:

The completion of the rolling submission for golodirsen includes data from the 4053-101 study assessing the safety, tolerability, pharmacokinetics and dystrophin expression of golodirsen in 25 boys with confirmed deletions of the DMD gene amenable to exon 53 skipping. The study demonstrated statistically significant results in favor of golodirsen on all biological endpoints, including properly exon-skipped RNA transcript using reverse transcription polymerase chain reaction, increase in quantity of dystrophin expression from baseline using Western blot and increase in dystrophin intensity as measured by immunohistochemistry.

If the golodirsen NDA is filed and granted accelerated approval, the company’s ESSENCE study (4045-301) could serve as a post-marketing confirmatory study. ESSENCE, which is under way, is a global, randomized double-blind, placebo-controlled study assessing the safety and efficacy of golodirsen and casimersen, our exon 45 skipping therapy.

Golodirsen is a phosphordiamidate morpholino oligimer engineered to treat those patients with Duchenne muscular dystrophy (DMD) who have genetic mutations subject to skipping exon 53 of the DMD gene. Patients with a 53 mutation represent 8 percent of those with Duchenne.

Click here to read the full press release.

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