Glob­al Blood Ther­a­peu­tic­s' sick­le cell dis­ease drug gets break­through ther­a­py sta­tus; Boehringer In­gel­heim will man­u­fac­ture BeiGene's PD-1 in­hibitor in Shang­hai

→ Glob­al Blood Ther­a­peu­tics just got break­through ther­a­py des­ig­na­tion from the FDA for its all im­por­tant sick­le cell dis­ease drug, vox­elo­tor (GBT440). The de­ci­sion was based on sev­er­al clin­i­cal pro­grams, in­clud­ing a PhI/II study in adults, an on­go­ing Ph2 tri­al in chil­dren, pre­lim­i­nary da­ta from a PhI­II study and com­pas­sion­ate ac­cess ex­pe­ri­ence. The BTD will help ex­pe­dite the re­view of vox­elo­tor, which is now the sole drug in the pipeline for the new­ly pub­lic biotech $GBT.

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