Accelerated approval of tofersen could galvanize neurology drug development
With FDA signaling it’s all-in on flexibility, signs look good for ALS therapy from Biogen, Ionis
While the signs look good for Biogen and Ionis heading into FDA’s review of tofersen, the ramifications could go well beyond the ALS therapy if neurofilament light chain gains status as a surrogate endpoint, providing a benchmark for the wide range of companies using the biomarker to track treatment response across the gamut of neurology indications.
Recent statements by FDA’s Office of Neuroscience about its intent to apply “maximum” regulatory flexibility to therapies for amyotrophic lateral sclerosis and its repeated willingness to adopt surrogate endpoints lacking clear scientific consensus appear to set the stage for an accelerated approval of tofersen, the antisense oligo from Biogen Inc. (NASDAQ:BIIB) and Ionis Pharmaceuticals Inc. (NASDAQ:IONS)...