FDA clears Glob­al Blood Ther­a­peu­tics to fo­cus on key bio­mark­er in piv­otal sick­le cell tri­al

Just days af­ter flesh­ing out its lat­est set of pos­i­tive da­ta from a tiny study of GBT440 for sick­le cell dis­ease, Glob­al Blood Ther­a­peu­tics $GBT says that the FDA has signed off on its piv­otal tri­al de­sign, agree­ing to stick with a key bio­mark­er for he­mo­glo­bin lev­els for the pri­ma­ry end­point, which in­ves­ti­ga­tors have al­ready suc­cess­ful­ly tried out in small­er tri­als.

Ear­li­er in Oc­to­ber, the biotech re­port­ed that among six pa­tients re­ceiv­ing treat­ment over 90 days there was a “me­di­an 1.1 g/dL (gram per deciliter) in­crease in he­mo­glo­bin con­cen­tra­tion with GBT440 treat­ment com­pared with a “0.2 g/dL de­crease with place­bo.”

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