FDA Adcomm Backs Accelerated Approval of Biogen’s Tofersen in SOD1-ALS

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1. Biogen and Ionis are closer to accelerated approval for tofersen in SOD1-ALS.

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2. FDA's advisory committee voted 9-0 that the drug's effect on NfL could predict clinical benefit.

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3. Traditional approval for tofersen was not recommended due to inconclusive data.

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4. Tofersen, licensed from Ionis in 2018, targets mutant SOD1 mRNA and lowers faulty protein levels.

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5. FDA accepted Biogen's New Drug Application for tofersen in July 2022.

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6. Phase III VALOR study didn't meet its primary endpoint, but showed reduction in SOD1 protein and NfL levels.

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7. FDA expressed concerns with relying on biomarker data for approval.

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8. Panelists disagreed, believing NfL could be a reasonably likely predictor of clinical benefit.

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9. If granted accelerated approval, Biogen must run a Phase III confirmatory study.

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10. The ongoing ATLAS study, with data expected in 2027, may fulfill the confirmatory study requirement.

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