The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Just another one on the regulatory interactions. Curious just in the discussions with the EMA and then also the BLA filing, has there -- have you
seen any differences in terms of how the U.S. versus EU is sort of viewing the submission? Any differences in terms of how they're viewing the
clinical data or endpoints?
Question: Kristen Brianne Kluska - Cantor Fitzgerald & Co., Research Division - Analyst
: So as there are multiple specialists often involved for the treatment of both Pompe and Fabry diseases, I wanted to ask, of your existing commercial
team for Galafold, what the overlap is for centers that also treat for Pompe disease. And then if you were to expand into more territories that AT-GAA
is approved, how do you believe this could also benefit the footprint for Galafold?
Question: Yun Zhong - BTIG, LLC, Research Division - Analyst
: So on AT-GAA, I think you mentioned that some patients, after long-term treatment with Lumizyme, they start to decline, losing response. And I
believe the reason is still not very well understood.
So I wonder -- I know it's too early to tell, but is there any evidence to suggest that this observation might not happen to AT-GAA? And in your
clinical study in the Pompe study, did you, by any chance, enroll any patients who might have entered that declining phase from receiving Lumizyme
treatment?
Question: Gil Joseph Blum - Needham & Company, LLC, Research Division - Analyst
: Congratulations on the execution. Maybe a bit of a question on the Batten disease program. Do you guys have any thoughts about this upcoming
AdCoM in September, which will discuss safety on AAV9 therapeutics?
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