FDA approves Takeda’s new enzyme replacement therapy for rare blood clotting disorder

The FDA on Thurs­day ap­proved Take­da’s Adzyn­ma, an en­zyme re­place­ment ther­a­py for adult and pe­di­atric pa­tients with con­gen­i­tal throm­bot­ic throm­bo­cy­topenic pur­pu­ra (cTTP), which is a rare and po­ten­tial­ly fa­tal blood clot­ting dis­or­der.

The ap­proval was based on da­ta an­nounced by Take­da in Jan­u­ary from an in­ter­im Phase III analy­sis that found the ther­a­py re­duced in­stances of low blood platelet count for pa­tients with cTTP com­pared to stan­dard-of-care plas­ma-based ther­a­pies.

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