Sarepta Therapeutics has announced that the FDA (the medicines regular in the US) has granted accelerated approval to golodirsen.
Golodirsen, or Vyondys 53 as it will be known, is an exon skipping drug designed to skip exon 53 of the dystrophin gene. It has the potential to treat approximately 8% of people living with Duchenne.
Trial data showed that Vyondys increased dystrophin production in the muscles of those who received it. Accelerated approval means the company can make the drug available while carrying out further trials to collect more data about the drug’s effectiveness.
This news is particularly exciting for us at Action Duchenne – the first trials of Vyondys 53 took place as part of the Skip-NMD project that we were heavily involved with alongside MDUK and PPMD Onlus. The project was an EU-funded collaborative effort between pharma, clinicians, researchers and charities to develop and test a drug to skip exon 53 of the dystrophin gene.
Because each country has its own medicines regulator, this decision won’t make the drug available to people outside the US. However, Sarepta are engaging with the European regulator to understand what data would be needed to support an application for marketing here. We hope the ongoing trials will provide the data required and we’ll be working with Sarepta to represent our families across the UK.
Wave halts development of exon skipping drugs
