Replies to post #24592 on Biotech Values

[rph_in_wi]

RE: the approval process has major shortcomings

When evaluating what went wrong, who do you blame for this rejection?

GTCB's loose dosing protocol, if not discussed with the EMEA beforehand, would be an immediate red flag. In the CC GTCB sounded like the need for repeated dosing adjustment was unexpected and that they now have a standard protocol. Their inability to understand the need for a standardized protocol may be due to the EMEA guidance or their own inexperience. I do understand that lives were saved by this dosing, but it is no way to run a study.

THe EMEA casts great suspicion on their motivations by the inclusion of some very obvious straw men (ie: filtration and antigenicity)in their rejection notice. As already discussed, these worries are weak and act only to draw attention away from a problem with the study group. The 2 year delay only reinforces the perception of ulterier motives and casts doubt on the integrety of the organization. Whether it is a discomfort with goat-derived medicine or a conspiracy to aid Pharming, the conspiracy folks certainly have the fodder.

So who dropped the ball?

RPh

[iwfal]

It’s easy for people to say that the whole two-year review process was BS and the application never had a chance to be approved for political reasons. I respectfully disagree. With more patients and no alteration in the dosing protocol, I think the application would have breezed through, politics and all.

We will never *know* for any drug whatsoever that it was rejected just for being a gmo. Unless someone confesses. Because drug applications are so enormously complex that you can *always* find something 'wrong'. What would it take to convince you that this was a political decision?

[aslan2772]

GTCB thoughts/politics

“It’s easy for people to say that the whole two-year review process was BS and the application never had a chance to be approved for political reasons. I respectfully disagree. With more patients and no alteration in the dosing protocol, I think the application would have breezed through, politics and all.”

I agree that things are not black and white, but IMHO GTCB is handicapped by the political aspects of transgenics, peppered with a bit of xenophobia. If anything goes wrong down the road with Atryn or subsequent drugs in this arena, the CHMP may want to have bullet-proof data to fall back on. If GTCB had bullet-proof data, things may indeed have turned out differently… but, on the other hand, if Atryn were derived from cultured milk glands (hypothetically speaking), the level of scrutiny might have been lower and the CHMP opinion may have fallen on the positive side. Thursday, I think GTCB investors saw (unfairly) a higher level of scrutiny applied for this novel (and fear-provoking for some) technology. CHMP or the EMEA ought to acknowledge this if indeed it is the case. Otherwise, it appears that GTCB management does not run a tight ship. Either way, it doesn't look very appealing.

On another note, this article may possibly be of interest: Post secretional modification? (sorry, I don’t have access to the article).
http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=pubmed&dopt=Abstract&list_...