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.’
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Accelerated Approval Is US FDA Panel’s Preferred Path For Biogen/Ionis’s Tofersen In ALS
Advisory committee unanimously concludes that the reduction in plasma neurofilament light chain concentration is reasonably likely to predict clinical benefit in SOD1-ALS, but majority of panelists say convincing evidence of efficacy to support regular approval has not been demonstrated.
Second Time’s The Charm: Amylyx’s ALS Drug Wins US FDA Panel Nod
In switching their votes from the first panel review in March, advisory committee members cited supportive additional analyses of a survival benefit, framing of the issues in the context of unmet need and regulatory flexibility, and the company’s statement that it would withdraw AMX0035 if the Phase III PHOENIX trial does not succeed.
Amylyx’s ALS Drug Lacks ‘Confirmatory Evidence’ Needed To Support Approval On Single Study, FDA Says
New analyses on individual responders, survival and biomarker data are not independent evidence of AMX0035’s clinical effect in ALS, agency says in advisory committee briefing document. However, FDA’s framing of the issues, with an emphasis on regulatory flexibility and unmet medical need, could sway a favorable recommendation in its second panel review.