2023-03-22 17:06:31 ET
- An FDA advisory panel voted 9-0 that the data on Biogen ( NASDAQ: BIIB ) and Ionis Pharmaceuticals ( IONS ) amyotrophic lateral sclerosis (ALS) drug tofersen likely predicts it benefit.
- The recommendation from the Peripheral and Central Nervous System Drugs Advisory Committee will now go before the agency for a final decision. The FDA usually, but not always, follows the recommendations of its advisory committees.
- 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.
- Although a pivotal trial missed its primary and secondary endpoints, FDA scientists said that biomarker readings indicated those biomarklers alluded to clinical benefit .
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Biogen ALS drug tofersen wins unanimous FDA panel recommendation