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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