Away from its adventures in Alzheimer’s disease, Biogen Pharma is hopeful of an accelerated US approval for tofersen, which it licensed from Ionis Pharmaceuticals, Inc., as the first treatment to target a genetic cause of amyotrophic lateral sclerosis (ALS).
On 22 March, the Food and Drug Administration’s Peripheral and Central Nervous System Drugs Advisory Committee decided that tofersen was suitable for an accelerated, but not regular, approval as the first disease-modifying treatment for superoxide dismutase 1 (SOD1) ALS, despite it missing the primary endpoint in the Phase III VALOR study
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