Sarepta Therapeutics eteplirsen update...

Muscular Dystrophy Campaign
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Thursday 25 July 2013

Sarepta Therapeutics eteplirsen update

In a press release, Sarepta Therapeutics yesterday announced that they plan to submit a licensing application for eteplirsen early in 2014. If the application is successful, Sarepta could be given permission to market eteplirsen in the USA. The company has also announced further details of a phase 3 trial being planned for next year and given an update on the results of the phase 2b trial that is ongoing.

In a press release, Sarepta Therapeutics yesterday announced that they have held recent meetings with the Food and Drug Administration (FDA), the drug regulator in the USA, in which they presented the latest results from their ongoing phase 2b trial of eteplirsen. Eteplirsen is a potential exon skipping drug for boys with Duchenne muscular dystrophy. Following these meetings, the FDA has said they would be willing to consider a licensing application based on the current trial results. Sarepta now plans to submit the application early in 2014.

A licensing application means that the FDA will review the evidence from clinical trials and pre-clinical testing of eteplirsen. They will decide whether the potential drug is safe and effective and decide whether Sarepta should be given a licence to market eteplirsen in the USA. It must be noted that this announcement is not a guarantee that the licence will be granted - just that the FDA is willing to look at an application. Also, the application will only apply to the USA - a separate application will need to be made in Europe. Sarepta is now working towards producing the application and will continue to meet with the FDA to make sure the application is as complete as possible.

Along with planning a license application, the company has started to scale up production of eteplirsen, a process which is so far going according to plan. Although the scaling up process will require a lot of testing and careful quality control, Sarepta hopes that by the end of 2014 it could be in a position to supply eteplirsen to between half and all of the boys in the USA who could benefit from the potential drug. If a license is not granted by the FDA, the increased production will be used to support a larger phase 3 trial which Sarepta is currently planning. This will aim to confirm the results of the current trial (see below for the latest results) and is likely to include approximately 50 boys each in a treatment and control group (who will not receive eteplirsen). The design of the trial is still being finalised and we will bring you more details when they become available. 

Recently, Sarepta also gave an update on the latest results of their ongoing phase 2b trial of eteplirsen. At a conference in Massachusetts, the company announced that the distance boys canwalk in six minutes is still stable after 84 weeks. Although this is encouraging, the trial includes only ten boys in total and so the results must be viewed with some caution. The company is still performing regular check-ups on all the boys in the trial to monitor the safety and effectiveness of the potential drug and plans to present results up to 96 weeks at a meeting of the World Muscle Society in October.