With tofersen approval, neurofilament becomes ‘anchor’ biomarker in ALS

FDA’s acceptance of neurofilament as a surrogate endpoint for ALS may not open the gates to a flood of approvals based on the surrogate endpoint, but it could give companies an alternative path to approval if their efficacy data in the notoriously heterogeneous disease turn out to be less than pristine.

The biomarker also stands to improve clinical trials for amyotrophic lateral sclerosis in other ways, such as helping create more homogeneous study populations, and could propel research into the next biomarkers by serving as a benchmark against which to test them...