Replies to post #1007 on GTC Biotherapeutics (fka GTCB)

[urche]

Factor VII uses


Monday’s CC gave the actual number of individuals in the indication that GTC and LFB plan to pursue—severe hemophilia with inhibitors: 21,000 in the U.S. and 36,000 in Europe. This works out to about 1 case in every 7,000 males.

Thanks for that, Dew. Interesting and important information, because this is a much bigger potential market pool than hereditary Factor VII deficiency, with incidence of only about 1:500,000. Perhaps we should view severe hemophilia with inhibitors as the most important acquired form of clotting disorder. My understanding is that this condition occurs in hemophilia patients who become immune to administration of standard treatment due to an immune mechanism. Factor VII, being later in the extrinsic pathway, is able to bypass that defect. I suppose the reason Factor VII is not used all the time is mostly expense.

Urche