FDA approves Biogen and Ionis’ Tofersen for rare genetic form of ALS

FDA approves Tofersen for rare genetic form of ALS

The FDA’s decision came just over a month after an advisory committee issued a mixed vote on Tofersen.

The FDA approved Tofersen to treat SOD1-mutation-mediated ALS based on a reduction in plasma neurofilament light, a blood-based biomarker of nerve injury and neurodegeneration. SOD1-mutation-mediated ALS affects approximately 330 people in the U.S. and is considered “uniformly fatal” by the National Institutes of Health.

“Today also marks a pivotal moment in ALS research as we gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS,” Biogen CEO Christopher A. Viehbacher said in a statement. “We believe this important scientific advancement will further accelerate innovative drug development for ALS.”