Biogen/Ionis’ Tofersen Wins Accelerated Approval In ALS Subgroup – A Significant But Narrow Precedent
US FDA designates ongoing ATLAS trial in presymptomatic individuals with SOD1 mutation as confirmatory study. ‘Classic Biogen’ approval is first in ALS under the expedited pathway and could lead to more drugs reaching market on basis of a reduction in plasma neurofilament light chain concentration.
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The European Medicines Agency is this week due to decide whether to back pan-EU marketing authorization for seven products, including three for orphan indications.
Promising Pathway Act Would Fill Gap For Rare Disease Treatments That Don’t Fit Accelerated Approval, Advocates Say
Bill would enable two-year provisional approval of drugs to treat life-threatening illnesses, such as ALS, based on substantial evidence of safety and early evidence of efficacy. At a Senate committee hearing, rare disease advocates said it would remedy the FDA’s inconsistent application of regulatory flexibility, but a bioethicist cautions it would lower the bar for approval.
After showing an impressive 60% reduction in tau levels in the brain, Biogen’s Phase I study of BIIB080 has now demonstrated early signs of an impact on cognition and daily living for Alzheimer’s patients.