Tale Of Two ALS Drugs: Similar Study Design, Different Take By US FDA

Amylyx’s AMX0035 and Mitsubishi’s edaravone showed similar treatment effect on ALS versus placebo in a single study, but the FDA found edaravone’s results to be more robust and supported by secondary endpoints.

Path fork
US FDA takes different paths in review of two ALS drugs • Source: Alamy

When the US Food and Drug Administration approved Mitsubishi Tanabe Pharma Corporation’s Radicava (edaravone) in 2017, it was the first treatment for amyotrophic lateral sclerosis to clear the agency in more than 20 years. The FDA is now considering Amylyx Pharmaceuticals, Inc.’s AMX0035 application for ALS, which is in some ways similar to that for edaravone. But the agency’s different views of the data show the difficulties in getting approval based on a single study.

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