FDA picks apart Brain­Stor­m's po­ten­tial ALS treat­ment ahead of Wednes­day ad­comm

The un­met need for pa­tients with the pro­gres­sive neu­rode­gen­er­a­tive dis­ease ALS, or amy­otroph­ic lat­er­al scle­ro­sis, is un­doubt­ed­ly high. But the FDA is draw­ing the line with Brain­Storm’s NurOwn (de­bame­stro­cel) in brief­ing doc­u­ments re­leased Mon­day ahead of Wednes­day’s ad­vi­so­ry com­mit­tee meet­ing of out­side FDA ex­perts.

The agency ex­plains the BLA for NurOwn that was filed over protest — a rare and of­ten des­per­ate move that spon­sors make when they have no oth­er op­tions — af­ter the spon­sor re­ceived a “refuse to file” let­ter from the FDA, when the com­pa­ny didn’t file the nec­es­sary da­ta in its orig­i­nal ap­pli­ca­tion.

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