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.
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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.
At Tofersen Panel Review, US FDA Shined A Light On Internal Differences Of Opinion
Agency’s presentations on Biogen/Ionis’ ALS drug included differing viewpoints from the statistical and clinical pharmacology teams; the transparency and emphasis on a multidisciplinary review approach may reflect lessons learned from missteps at the Aduhelm panel review in 2020.
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.'