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# Panel Backs Accelerated OK Consideration of Biogen's ALS Drug
- URL: https://www.fdaweb.com/panel-backs-accelerated-ok-consideration-of-biogens-als-drug/
- Published: 2023-03-23T12:00:00.000Z
- Updated: 2026-09-14T18:21:51.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5154049

FDA’s Peripheral and Central Nervous System Drugs Advisory Committee 3/22 voted 9 to 0 that Biogen’s amyotrophic lateral sclerosis (ALS) drug tofersen should be considered for accelerated approval. Panel members agreed that a biomarker assessed in tofersen’s clinical trial — a reduction in plasma neurofilament light chain (NfL) —is reasonably likely to predict clinical benefit in treating patients with the superoxide dismutase 1 (SOD1) gene, which is associated with 20% of familial cases and about 2% of sporadic ALS cases. The company proposed the accelerated approval pathway after the drug’s primary study failed to show a statistically significant difference from placebo groups for the primary and secondary endpoints.

A second vote that asked the panel whether the totality of data could support a traditional approval was met with significantly less support, with panel members voting 5 to 3 (one abstention) that the data do not justify such a move, mainly due to the primary trial missing the endpoints.

In voting yes for traditional approval, Abington Neurologic Associates Clinical Research Center director **David Weisman** said he agonized over his decision but the “odds are extremely high that this drug works. And I guess I really worry about the consequences of an accelerated approval, maybe as a neurologist, where payers will not cover this drug and consider it experimental, which I think would be a big problem clinically.”

Wake Forest University School of Medicine professor **Michelle Mielke** also voted yes for traditional approval and noted that while all of the data are not fully conclusive, there are “several aspects of the data that do suggest strong clinical evidence. My decision also weighed in the fact that this really is an unmet need and ALS is a very serious disease. So the fact that some people may be stalling in terms of their progression or possibly improving is very promising.”

After the panel meeting, Biogen said it was moved by the positive support of the ALS community during the open public comments period and it remains hopeful that: “(i)f approved, tofersen would potentially represent a major advance for people living with SOD1-ALS.” FDA is expected to issue a review decision by 4/25.