Replies to post #10344 on GTC Biotherapeutics (fka GTCB)

[DewDiligence]

>you are both using the term "strong data" to defend your views on the likelihood of an advisory panel for ATryn<

Actually, I did not render an opinion on the likelihood that the FDA will convene a panel.

[palindromy]

I agree the data is only strong from the relative viewpoint of this is what the FDA wanted and this is what they got. Of course the FDA is well-known for sticking to their goalposts!

The discussion of transgenic stuff and implications will happen anyway. I would rather it happen in a public forum rather than within the FDA itself amongst bureaucrats, especially risk-averse ones.

While I think the overall approval chances are strong in either case, it becomes stronger if there is a public discussion rather than private.

[vinmantoo]

Quantum,

<My only caution is that although the data points they have are good (safety, equivalence etc.,) they are very few in number. I know this is in a very small patient population and larger trials were not commercially feasible.>

I can't rule out an advisory panel. But you have to keep in mind that GTCB worked with the FDA to design the phase III trial and it was decided then how many patients were need for GTCB to generate sufficient data to merit approval. The phase III trial utilized the requisite number of patients and passed with flying colors. This makes the idea that there were too few patients a bit of a red herring. I do agree that an advisory panal still cannot be ruled out given that rATIII will be the first transgenic protein approved. Howver, I must agree with Dew, I would be far more confident of approval if no panel was convened.