First Treatment for Genetic Form of Amyotrophic Lateral Sclerosis Approved by FDA

The Food and Drug Administration (FDA) approved the first treatment for amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. Tofersen (QALSODY; Biogen, Cambridge, MA) 100 mg/15 mL injection was approved under accelerated approval based on results of a study documenting reductions in plasma neurofilament light chain levels seen in those treated with tofersen. SOD1-ALS is an ultra-rare, fatal genetic disease affecting approximately 300 individuals in the United States. ATLAS, an ongoing Phase 3 study, will continue as a confirmatory trial for tofersen in people who are presymptomatic for SOD1-ALS.

Christopher A. Viehbacher, President and Chief Executive Officer of Biogen noted that this announcement “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. We believe this important scientific advancement will further accelerate innovative drug development for ALS.”

There are important warnings and precautions associated with treatment with tofersen including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis. The most common adverse reactions reported in > 10% of participants treated with tofersen included pain, fatigue, arthralgia, cerebrospinal (CSF) white blood cell increase and myalgia.