What is the FDA comparing to?

Posted by: Christine McSherry on December 19, 2013


The results from the GSK Phase 3 trial were released. What I found discouraging is that Baseline muscle biopsies were never done, therefore, very difficult to measure dystrophin or lack of Ė I have to wonder what the FDA will find to support eteplirsen in these results. How can the FDA compare two drugs or the mechanism of action of two drugs, when you canít confirm the absence of dystrophin in the drisapersen trial participants?

Results reported from GSK Phase 3, read here.

There are lots of questions here, but few answers.† As a community, we need to be concerned that a potentially effective treatment, such as eteplirsen, may become victim to the failures or shortcoming over other drugs or drug trials.† More troubling is whether the FDA is making conclusions about these drugs and their ability to produce dystrophin without either the data or expertise to analyze it and understand it.† We have a lot riding on the FDAís ability to understand these issues, to insure that they are not making uninformed or less than informed assumptions and that ALL data that may support the research community understanding these issues be released in detail.† We donít have time to waste and their certainly should be no patience or tolerance for companies who do not provide transparency and access to needed data so that we can answer these questions and insure that the FDA and others are using the best information possible as they make important, life altering/saving decisions, including their near term decision on eteplirsen.† Please join me in asking Glaxo, Prosensa and PTC to put forward the biopsy data they have collected from over 500 boys so that our community, our families and the FDA can make informed decisions based on real data.

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