One Difference Between Amylyx’s ALS Drug And Aduhelm: A Biomarker Endpoint

US FDA’s Billy Dunn says that if the drug had shown a positive effect on plasma neurofilament heavy chain, it ‘would have provided important contextual and supportive information of an ostensibly beneficial effect on the clinical measure.’

ALS
Biomarker data could have helped show efficacy of Amylyx's ALS drug AMX0035 • Source: Alamy

Amylyx Pharmaceuticals, Inc.’s drug AMX0035 for treatment of amyotrophic lateral sclerosis will not be following the approval pathway of Aduhelm, at least not based on a surrogate endpoint. US Food and Drug Administration officials noted at an advisory committee meeting that there is no established biomarker for AMX0035 but that it would have been helpful to “the contextual picture” if AMX0035 had been shown to have an impact on a promising potential biomarker.

The agency granted accelerated approval for Biogen, Inc./Eisai Co., Ltd.'s Aduhelm (aducanumab) based on a single positive Phase...

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