GTC Biotherapeutics (fka GTCB)

[DewDiligence]

>Any idea which [FoB's] will require more detailed clinicals?<

Let’s consider the classes in #msg-20699044 on a case by case basis.

Insulin, hGH, and FSH are already regulated by the FDA as small molecules, so a new regulatory pathway is not needed to bring FoB’s to market for these classes. In theory, that is. In practice, as we saw with NVS’ Omnitrope (hGH), the FDA fought its approval tooth and nail and ended up approving it only because NVS sued and beat the FDA in federal court.

Still, insulin, hGH, and FSH are simple, well-understood proteins. I would not expect FoB’s from these classes to need more than a token clinical program to get FDA approval. It ought to be even easier in the EU.

For EPO, I agree with you that fairly extensive clinical trials will likely be needed. The sheer magnitude of this class and the high-profile safety issues that have arisen in the past will make it hard for the FDA to cut any corners here.

I think G-CSF will probably go the same way as EPO.

FoB’s for the enzyme-replacement drugs sold by GENZ and others will probably need full-fledged clinical programs because the drugs are taken for life and even a relatively minor deficiency in a FoB could have calamitous consequences. Moreover, the number of patients taking these drugs is too small for there to be a major political imperative to lower prices.

Although tallied as a single class in the table in #msg-20699044, the anti-cancer mAbs are highly disparate and do not really constitute a class from a scientific standpoint. Each of these drugs (Rituxan, Herceptin, Avastin, Erbitux, etc.) will have to be evaluated on its own merits.

What’s left? The TNF-a mAbs, the recombinant plasma proteins, and the interferons (alpha and beta). These are the classes that I think are best suited to abbreviated clinical programs for FoB’s that rely to a considerable extent on analytics as a substitute for some of the usual clinical work.

JMHO, FWIW