FDA again finds Amy­lyx's ALS da­ta lack­ing ahead of un­usu­al sec­ond ad­comm, while stress­ing dire need for treat­ments

The FDA’s brief­ing docs are in ahead of its un­usu­al sec­ond ad­comm for Amy­lyx Phar­ma­ceu­ti­cals’ ex­per­i­men­tal ALS drug next week — and the agency’s opin­ion is large­ly neg­a­tive.

While ac­knowl­edg­ing that ALS treat­ments are “des­per­ate­ly need­ed” and pro­vid­ing an ex­ten­sive his­to­ry of their pre­vi­ous flex­i­bil­i­ty for the dis­ease, FDA re­view­ers wrote that Amy­lyx’s sub­mis­sion of new analy­ses is “not in­de­pen­dent da­ta and is sim­ply a new method for an­a­lyz­ing the same sur­vival da­ta pre­sent­ed in the orig­i­nal NDA sub­mis­sion.” The new da­ta pack­ages in­volve pa­tients who switched over from treat­ment to place­bo af­ter the Phase II tri­al wrapped up, sug­gest­ing bet­ter sur­vival re­sults than the study orig­i­nal­ly in­di­cat­ed.

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