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.
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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.
Oral or feeding tube administration of the cerebral neuroprotectant should offer flexible dosing options for amyotrophic lateral sclerosis patients, the firm says.
Six of 10 advisory committee members say Phase II CENTAUR trial cannot stand on its own to support approval at this time. However, patients and advocates say thousands will die if they have to wait for the Phase III PHOENIX trial to complete in 2024 before the drug becomes widely available.