Sarepta applies for approval of second Duchenne drug

Published Date
Jenny Sharpe
Human DNA

Sarepta Therapeutics has filed an application with the US Food and Drug Administration (FDA) seeking approval of golodirsen (SRP-4053). If successful, this will be Sarepta’s second exon skipping drug approved by the FDA (after Exondys 51).

Golodirsen is an antisense oligonucleotide (or molecular patch) that binds to exon 53 of the dystrophin gene and triggers exon skipping. This could be a potential treatment for about 8% of people with Duchenne muscular dystrophy.

Sarepta has submitted data from its 4053-101 study to the FDA. This study included 25 participants and showed that golodirsen induced exon 53 skipping in the muscle, which led to an increase in dystrophin.

Sarepta has applied for accelerated approval, which allows fast approval of drugs for serious conditions with an unmet need. If its application is successful, the company’s ongoing ESSENCE study (4045-301) could be used as a post-marketing study to confirm golodirsen’s effectiveness.

Although this is good news, the FDA regulates drugs in the USA and not in Europe. We hope that Sarepta will announce its plans for Europe very soon.

For more information, read Sarepta’s press release.

If you have any questions about this news story or other Duchenne research, please contact the MDUK Research Line on 020 7803 4813 or email

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