Photo: Manuel Balce Ceneta/AP Images

FDA ad­vi­sors vote 6-4 against Amy­lyx's ALS drug, fur­ther dim­ming its hope for reg­u­la­to­ry suc­cess

In a marathon hear­ing Wednes­day, out­side FDA ad­vi­sors rec­om­mend­ed to the agency that a sin­gle ran­dom­ized study did not pro­vide con­clu­sive proof over whether an ex­per­i­men­tal ALS drug is ef­fec­tive.

Pan­elists vot­ed 6-4 in the neg­a­tive, sub­stan­ti­at­ing the agency’s con­cerns about the ro­bust­ness of da­ta in a Phase II study run by Amy­lyx Phar­ma­ceu­ti­cals — by the slimmest pos­si­ble mar­gin. The drug, known as AMX0035, pro­duced a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion on a func­tion­al rat­ing scale, but the ef­fect proved un­con­vinc­ing to the com­mit­tee.

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