Sarepta therapeutics has today announced that they have completed the submission of a USA application for  accelerated approval of golodirsen – a drug designed to skip exon 53 of the dystrophin gene, which the company believes might help 8% of people living with Duchenne.

The New Drug Application (or NDA), which has been submitted to the FDA (the drug regulator in the US) includes data from a clinical trial that included 25 participants and assessed the safety and tolerability of the potential drug as well as the production of dystrophin in the muscles. Golodirsen induced exon skipping as expected and an increase in dystrophin production was also measured. Based on these initial results, Sarepta has applied for accelerated approval, and has already started further trials to test the effectiveness of golodirsen in a larger group of boys.

If golodirsen gains accelerated approved, it will only be for the USA – in the European Union drugs are approved by the European Medicines Agency. Sarepta has not yet announced plans to apply for approval in Europe, but the submission of the application in the USA shows that their exon skipping programme continues to move forward.

Find out more

Contact Neil Bennett, Director of Research, to find out more on 020 7250 8240

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