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.

Pipes misaligned
The FDA statistical and clinical pharmacology teams' respective views on tofersen were not in alignment. • Source: Shutterstock

The US FDA Office of Neuroscience’s presentation approach at the recent advisory committee review of Biogen, Inc. and Ionis Pharmaceuticals, Inc.’s Qalsody (tofersen) for SOD1 amyotrophic lateral sclerosis appears to have reflected the hard lessons learned from the November 2020 Aduhelm (aducanumab-avwa) panel meeting.

In a situation where there was clearly internal disagreement among review disciplines – specifically, the statistical team on one side,...

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