Armed with up­dat­ed 24-week PhI­II da­ta, GBT con­vinces FDA to re­view sick­le cell drug un­der ac­cel­er­at­ed path­way

In yet an­oth­er sign of the FDA’s en­hanced flex­i­bil­i­ty un­der com­mis­sion­er Scott Got­tlieb, Glob­al Blood Ther­a­peu­tics $GBT has man­aged to con­vinced the reg­u­la­tor to al­low its ex­per­i­men­tal drug, vox­elo­tor, to be eval­u­at­ed un­der the ac­cel­er­at­ed ap­proval path­way for sick­le cell dis­ease (SCD), a group of in­her­it­ed red blood cell dis­or­ders that typ­i­cal­ly af­flict those of African an­ces­try.

SCD im­pacts he­mo­glo­bin, a pro­tein found in red blood cells that car­ries oxy­gen through­out the body, and is char­ac­ter­ized by episodes of sear­ing pain as well as or­gan dam­age.

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