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Biogen/Ionis’ Tofersen Wins Accelerated Approval In ALS Subgroup – A Significant But Narrow Precedent

 
• By Sue Sutter

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.

Footsteps on beach
Other ALS drug developers will look to follow Biogen/Ionis' lead in using reduction in neurofilament concentration as the basis for accelerated approval. • Source: Shutterstock

The US Food and Drug Administration opted for accelerated approval, rather than regular approval, for Biogen, Inc. and Ionis Pharmaceuticals, Inc.’s Qalsody (tofersen) in a genetically defined subset of amyotrophic lateral sclerosis patients, banking that clinical benefit will be confirmed in an ongoing trial of presymptomatic individuals.

The agency approved tofersen, an antisense oligonucleotide, on 25 April for treatment of ALS in adults who have a mutation...

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