Replies to post #24597 on Biotech Values

[Biowatch]

GTCB - I still do not understand why GTCB apparently only just found out that the pregnant patients could not be included in the evaluable patient data base subject to review in the clinical trial, and if they did not count, or could not count, why this issue was not raised in advance.

And yes, if GTCB is going to get blind-sided like this by the FDA, they better find out now.

As I mentioned previously, it's a lot easier to figure out when you are pregnant than to know when you will need surgery, so it is presumably easier to recruit pregnant patients than surgical patients for these trials.

If they have to get separate approval for pregnant patients with this extremely rare disease and for surgical patients with this extremely rare disease, then it seems like a miniscule market, even if it saves lives and, in the case of pregnant patients, leads to healthy children without the disease.

[DewDiligence]

>When evaluating what went wrong, whom do you blame for this rejection?<

65% GTC, 25% EMEA staff, and 10% CHMP. This is based on the assumption that the EMEA staff’s recommendation to the CHMP was not uniformly negative.

I think GTC deserves most of the blame for muddying the trial design in order to complete the pivotal trial sooner. In clinical trials, simpler is almost always better.

Until today, I took it as a given that it was reasonable to combine pregnancy and elective surgery into one study, but clearly that was not the case. GTC evidently did not anticipate the difficulties in such a combination, but they ought to have anticipated such difficulties, IMO.

The EMEA deserves a share of the blame because, knowing that there were issues with the dosing protocol and the number of surgical patients, they ought to have informed GTC in 2004 that more surgical patients needed to be tested. The results of such testing could have been completed and analyzed by now.

The CHMP deserves a small slice of the blame because they had an opportunity to approve ATryn despite the EMEA’s misgivings. It’s a pretty good bet that ATryn is as safe and effective as plasma-derived antithrombin and that more testing will not fundamentally change the basis for making an informed decision on approvability.

If Atryn were a fundamentally new drug, I would have said that the CHMP did the right thing to reject given the doubt by the EMEA staff. However, ATryn is just antithrombin produced differently. The impurities are negligible (below the detection levels of the assays), and there has been no evidence of immunogenicity in 200+ ATryn recipients in various clinical trials. It ought not to have mattered to the CHMP that some of these 200+ recipients were not patients undergoing elective surgery because immunogenicity is unlikely to be dependent on the specific medical indication for which ATryn is given. Thus, the CHMP had an opportunity to make a science-based decision to overrule the EMEA staff.

JMHO, FWIW