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Accelerated Approval: Potential Sources Of Confirmatory Evidence Weighed At Tofersen Review

 
• By Sue Sutter

‘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.'

Eggs in nest
Biogen has proposed several sources of confirmatory and supportive evidence for tofersen's clinical benefit. • Source: Shutterstock

Potential sources of confirmatory evidence were on the minds of US Food and Drug Administration advisory committee members at their recent review of Biogen, Inc. and Ionis Pharmaceuticals, Inc.’s Qalsody (tofersen) for amyotrophic lateral sclerosis patients with a specific genetic mutation.

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