Biogen’s ALS Drug Receives Unanimous Recommendation Accelerated Approval from FDA Adcomm

March 23, 2023

Tofersen, Biogen’s ALS Drug, has received a unanimous 9-0 recommendation for accelerated approval by the US Food and Drug Administration’s Peripheral and Central Nervous System Drugs Advisory Committee. The move comes in spite of results from a Phase III trial in which the drug failed to meet its primary outcome of reducing disease activity from baseline at 6 months. It did, however, reduce levels of a key protein in ALS pathophysiology, which the committee believes may impart long-term benefits.

According to Max Gelman, “Tofersen is an antisense oligonucleotide designed to treat ALS patients who possess a mutation of the SOD1 gene. While SOD1 is the second-most common ALS genetic mutation among those with hereditary disease, the group makes up only about 1% to 2% of all ALS patients. In the US, that amounts to about 300 people, with 100 patients newly diagnosed each year. Such a small patient population makes it far more challenging to run another placebo-controlled trial.”

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