Today marks a much anticipated major milestone in the treatment of Duchenne, as Translarna™ (ataluren) was granted conditional marketing authorization in the European Union for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. This is the first approved treatment to target one of the primary causes of the disease and it is a landmark moment for the company, patients and advocacy organizations alike. The drug will treat approximately 13% of Duchenne patients whose disease is caused by a nonsense mutation.
The company is obligated to complete a confirmatory Phase 3 trial in nonsense mutation Duchenne patients and submit additional efficacy and safety data. That trial is planned to enroll 220 patients at approximately 50 investigational sites globally and is currently recruiting. For further information, see https://www.clinicaltrials.gov/ct2/show/NCT01826487?term=ataluren&rank=8.
This approval comes at a busy time in Duchenne drug development. Prosensa and Sarepta Therapeutics are each anticipated to file an NDA later this year for market authorization in the US for their product candidates that induce exon 51 skipping in the dystrophin gene and is intended to treat approximately a further 14% of Duchenne patients.