US Food and Drug Administration reviewers appear unpersuaded that Amylyx Pharmaceuticals, Inc. has provided the confirmatory evidence needed to support approval of its amyotrophic lateral sclerosis drug AMX0035 on the basis of a single Phase II trial.
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.
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This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
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The European Medicines Agency is set to issue opinions this week on whether pan-EU marketing approval should be granted to a number of new products.