Biotech Values

[iwfal]

No, as pre-condition of Fabrazyme AA by FDA, a larger and longer duration randomized placebo controlled trial in similar but not identical population was conducted in many countries including US and EU where Fabrazyme was actually granted approval more than one year earlier than US under EMA's exceptional circumstances along with Shire's Replagal.

Yes - and that trial completed in approx early 2004 as best I can tell. And had probably already completed enrollment at the time of approval. I.e. The company knew enrollment wasn't going to happen once approved.

FDA rarely if ever requires confirmatory trial in exactly same population as AA.

I'd bet a whole lot of $ that few of those incensed about "the inflexible FDA" were thinking that some kids would be left off the AA label so *they* could be the lucky ones to support an RCT.