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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