Positive pivotal data in hand, GBT is hustling to the FDA in search of a quick OK for sickle cell drug -- but that's no easy task
Global Blood Therapeutics surprised some observers this morning with their plan to take the first round of positive late-stage data for the sickle cell disease drug voxelotor straight to the FDA in search of an accelerated approval.
The regulatory strategy is based on their data for the primary outcome of the study, with 58% of the patients in the high dose of the arm demonstrating a 1 g/dL-plus increase in hemoglobin at 12 weeks compared to only 9% of patients taking a placebo. In the lower-dose arm there was a 38% response rate, passing the bar set by the FDA.
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