FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A pan­el of out­side ad­vi­sors to the FDA unan­i­mous­ly rec­om­mend­ed that the agency grant ac­cel­er­at­ed ap­proval to Bio­gen’s ALS drug tofersen de­spite the drug fail­ing the pri­ma­ry goal of its Phase III study, an en­dorse­ment that could pave a path for­ward for the treat­ment.

By a 9-0 vote, mem­bers of the Pe­riph­er­al and Cen­tral Ner­vous Sys­tem Drugs Ad­vi­so­ry Com­mit­tee said there was suf­fi­cient ev­i­dence that tofersen’s ef­fect on a cer­tain pro­tein as­so­ci­at­ed with ALS is rea­son­ably like­ly to pre­dict a ben­e­fit for pa­tients. But pan­elists stopped short of ad­vo­cat­ing for a full ap­proval, vot­ing 3-5 against (with one ab­sten­tion) and large­ly cit­ing the failed piv­otal study.

Endpoints News

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.