Accelerated Approval: Potential Sources Of Confirmatory Evidence Weighed At Tofersen Review
‘Clear signal’ in the ATLAS study of presymptomatic, SOD1 mutation carriers could provide evidence needed to confirm tofersen’s benefit in symptomatic ALS patients, FDA’s Teresa Buracchio says, adding that getting confirmatory evidence from the VALOR open-label extension 'could be a little more tricky.'
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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.
Accelerated Approval Is US FDA Panel’s Preferred Path For Biogen/Ionis’s Tofersen In ALS
Advisory committee unanimously concludes that the reduction in plasma neurofilament light chain concentration is reasonably likely to predict clinical benefit in SOD1-ALS, but majority of panelists say convincing evidence of efficacy to support regular approval has not been demonstrated.
Biogen/Ionis’ Tofersen: US FDA Considering Both Accelerated And Regular Approval In ALS
FDA seeks advisory committee input on whether there is ‘convincing evidence’ to support regular approval for treatment of SOD1-ALS patients even though lone Phase III trial failed its primary endpoint; companies filed for accelerated approval on the basis that a reduction in plasma neurofilament light chain is reasonably likely to predict clinical benefit.