Duchenne exon skipping drug approved by FDA

Published Date
13/12/2019
Author
Louise Clarke
Category
Research
Orange box stating: Breaking news research

Sarepta’s exon skipping drug, VYONDYS 53™ (golodirsen) has been approved by the Food and Drug Administration (FDA). The FDA regulates drugs in the USA.

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

Read Sarepta Therapeutics press release.

Read about Sarepta’s ESSENCE study

 

Previous news related to this story:

 

20 August 2019

The US Food and Drug Administration (FDA) has issued a Complete Response Letter to Sarepta Therapeutics in relation to its application for approval of Duchenne drug golodirsen (also known as Vyondys 53 and SRP-4053). This means that the FDA cannot approve golodirsen based on the data submitted. Sarepta has said it will “immediately request a meeting with the FDA to determine next steps”.

The safety and efficacy of golodirsen is currently being evaluated in a global phase 3 clinical trial called ESSENCE. The rejection from the FDA should not affect participants receiving golodirsen as part of the ESSENCE trial.

For more information, read Sarepta’s press release.

3 January 2019

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.

12 March 2018

Sarepta Therapeutics has announced that it will file an application with the FDA by the end of 2018 seeking approval for golodirsen (SRP-4053). Golodirsen is an exon skipping drug that targets exon 53 of the dystrophin gene. Sarepta recently met with the FDA to ask for regulatory guidance on golodirsen. The FDA said that accelerated approval could be obtained if Sarepta proves that the drug can significantly increase dystrophin production in people with Duchenne. This has already been demonstrated in the 4053-101 study, which Sarepta intends to submit as part of its application.

For more information, read Sarepta’s press release.

6 September 2017

Sarepta Therapeutics has announced the latest results from the 4053-101 study (see SKIP-NMD project for more details). This phase 1/2 clinical study is assessing the safety and efficacy of golodirsen, a drug that skips exon 53 in individuals with Duchenne muscular dystrophy. Muscle biopsies from the treated participants showed that the drug was able to skip exon 53. This had a positive effect on dystrophin production.

Please read Sarepta Therapeutics’ press release for more information

If you have any questions please contact our research team at research@musculardystrophyuk.org or call 020 7803 4813

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