Biogen ALS drug tofersen earns mixed voting by FDA advisory panel
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Editor's note: This article was updated at 520p to reflect a second vote from the panel.

An FDA advisory panel gave mixed signals Wednesday afternoon in two votes regarding Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (IONS) amyotrophic lateral sclerosis (ALS) drug tofersen.

By a 9-0 vote, the Peripheral and Central Nervous System Drugs Advisory Committee voted that a reduction in a biomarker likely predicts clinical benefit of the drug.
However, in a later vote, the same panel voted 5-3 with one abstention that data from a placebo-controlled trial, and extension study, along with the biomarker data, did not provide convincing evidence of the benefits of tofersen.
Although a pivotal trial missed its primary and secondary endpoints, FDA scientists said that biomarker readings indicated those biomarkers alluded to clinical benefit.
The votes from the Peripheral and Central Nervous System Drugs Advisory Committee will now be discussed by the FDA who will make a final decision.
Tofersen is intended for people with a rare form of ALS that shows mutations in the superoxide dismutase 1 (SOD1) gene. This accounts for ~20% of familial cases.