FDA Accelerated Approval of Tofersen Highlights Importance of Blood Neurofilament Light Chain as Surrogate Endpoint in Neurology Therapeutic Trials

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Apr 25, 2023

Quanterix Corporation (NASDAQ: QTRX), a company fueling scientific discovery and breakthrough diagnostics through ultrasensitive biomarker detection, today announced that blood-based NfL measurements provided compelling support for the FDA’s accelerated approval of tofersen for treatment of superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS), a devastating rare genetic form of ALS. This is the first known case in which a blood biomarker was successfully used as a surrogate endpoint for a neurology therapeutic trial to gain accelerated approval, highlighting the potential for other therapeutic trial designs to benefit from including blood NfL measurements.