Saturday, October 01, 2011 2:59:49 PM
BMRN/Pompe:
In the webinar they indicated the FDA wouldn't let them go directly to trying it on younger patients with an already approved therapy in that population. The implication being it wasn't ethical to try an unproven therapy in kids when an approved option already exists. It makes sense to me now that I know
. Interestingly BMRN never directly came out and said as such at least not to my recollection. I think it was geared to families/clinicians to some degree. A father indicated he'd be more then willing to discontinue therapy (I assume Myozyme) for some time if it meant being able to get his child in a trial with it.
In the webinar they indicated the FDA wouldn't let them go directly to trying it on younger patients with an already approved therapy in that population. The implication being it wasn't ethical to try an unproven therapy in kids when an approved option already exists. It makes sense to me now that I know
. Interestingly BMRN never directly came out and said as such at least not to my recollection. I think it was geared to families/clinicians to some degree. A father indicated he'd be more then willing to discontinue therapy (I assume Myozyme) for some time if it meant being able to get his child in a trial with it.
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