Pharmamarketeer
FDA approves Tofersen for rare genetic form of ALS

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.”

Medhc-fases-banner
Advertentie(s)