Tofersen panel bolsters case for neurofilament as ALS surrogate endpoint 

Neurofilament appears on track to become the first surrogate endpoint for ALS after an advisory committee unanimously voted in favor of accelerated approval of tofersen based on the biomarker. However, a full approval of the therapy based on current data would be a bridge too far, according to the panel.

The Peripheral and Central Nervous System Drugs Advisory Committee voted 9-0 in favor of plasma NfL concentrations being reasonably likely to predict clinical benefit due to tofersen treatment in amyotrophic lateral sclerosis patients with SOD1 mutations. ...