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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER