2023-03-22 18:05:49 ET
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.
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Biogen ALS drug tofersen earns mixed voting by FDA advisory panel