"We've made a lot of preparations for what that confirmatory program could be, but we don't want to initiate enrollment into a confirmatory study until we're sure that we have the issues that are in consideration addressed in a confirmatory study. Now I should also remind you that we have ongoing a very large natural history study, reported some preliminary results to that in April at the American Association of Neurology. So we're already accumulating prospective natural history data on patients. And we clearly have been in discussion with the Food and Drug Administration about the nature of next steps of studies. We don't have anxiety about where we stand in regard to pulling the trigger, if you will, on a confirmatory study."
Biomarin CEO Jean-Jacque Bienaime added, " And if may, I mean, correct me if I'm wrong, Hank. But I mean, so far, we've never heard from the FDA that inititation of the confirmatory trial was a prerequisite for approval of the drug."
To which Fuchs replied, "That's correct."The FDA's June 2014 letter to Prosensa suggests otherwise. The controversy will be cleared up on Nov. 24.
A complete copy of the FDA letter written to Prosensa in June 2014 can be found here.