FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednes­day’s ad­vi­so­ry com­mit­tee hear­ing to dis­cuss Bio­gen’s ALS drug tofersen, the FDA ap­peared open to ap­prov­ing the drug, new­ly re­leased brief­ing doc­u­ments show.

Cit­ing the need for flex­i­bil­i­ty in a dev­as­tat­ing dis­ease like ALS, reg­u­la­tors sig­naled a will­ing­ness to con­sid­er green­light­ing tofersen based on its ef­fect on a cer­tain pro­tein as­so­ci­at­ed with ALS de­spite a failed piv­otal tri­al. The doc­u­ments come af­ter reg­u­la­to­ry flex­i­bil­i­ty was part of the same ra­tio­nale the agency ex­pressed when ap­prov­ing an ALS drug last Sep­tem­ber from Amy­lyx Phar­ma­ceu­ti­cals, in­di­cat­ing the FDA’s open­ness to ap­prov­ing new treat­ments for the dis­ease.

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