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.