Reading Dr. Temple’s Transcript from PPMD Conference…

Posted by: Christine McSherry on July 17, 2013

As I stated earlier, while the FDA is reviewing eteplirsen for approval, and many of us are sitting and waiting…it gives me and others, comfort to read, review, speculate on every detail found relating to accelerated approval.

Here is a quote that I took from Dr. Temple’s presentation that he gave during the PPMD conference in June.  Dr. Temple states that there is a production of a “Becker-type dystrophin”.  Does this mean that he believes it is being produced – so the only question left is whether or not it can be used as a surrogate endpoint?  Am I wrong, or did he answer his own question?

“But it’s perfectly obvious from discussions that have gone around that apart from the question

of whether the production of a Becker-type dystrophin will be an acceptable surrogate, it’s not

decided yet I’m not telling you anything one way or the other, for initial approvals, we will

certainly consider such issues, whatever we decide there, with respect to subsequent drugs for

different genomic lesions because success on a clinical endpoint might very well support that

kind of surrogate endpoint.”

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