Additional urgency for accelerated approval of eteplirsen from parents.

Posted by: Christine McSherry on September 24, 2013


As one of parents who have been advocating for a faster pathway of eteplirsen – there are several major points that we feel need to be communicated to the Duchenne community and the FDA.  I believe that there could be data pulled from drisapersen study that could strengthen and enhance the argument for accelerated approval for eteplirsen.

  • Eteplirsen produces a truncated – Becker type protein in 100% of patients at levels expected to result in clinical benefit, this has been demonstrated in the recent data presented. We know that many Becker patients have a longer survival and slower progression of the disease than Duchenne.

  • The drug appears safe, even after 2+ years, there have been no reported side effects.

  • More data, all would benefit from additional data — there are 2 ways to get this: small clinical trials which limits the number of patients who can be on the drug or Accelerated Approval, which gives all boys a chance and gets us the answers more quickly – accelerated approval.

  • FDA has asked for a 4th biopsy, a painful invasive, risky surgery — not clear what they will do with those results, the community and parents need FDA’s input here.

  • Last week, a phase III trial of a different drug, drisapersen, failed to meet clinical endpoints. Drisapersen’s toxicity limits the drug’s ability to overcome the variability amongst patients that has been seen in numerous low-dose exon skipping studies. Eteplirsen’s safety profile allows it to be administered at higher doses, therefor it has no such limitation.


The outcome and natural history of Duchenne is known. The outcome is always the same.  Those diagnosed who start to decline – don’t get better, ever. By not doing anything – by delaying a potential therapy to those who are sentenced to death, is worse than doing something.  The parents have told the FDA that they are willing to take permissible risk.  The FDA has all the information it needs to make an informed decision. The data stands on its own, as do 10 of the boys in the trial — and 2 young guys who still have tremendous upper body strength – if there were ever a time to be lucky when diagnosed with Duchenne – being included in the eteplirsen trial is lucky.

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