Early PHOENIX Data Would Not Have Aided Approval Decision On Amylyx’s Relyvrio – US FDA
Agency considered Amylyx’s plan to submit unblinded efficacy data from the ongoing Phase III trial during the ALS drug's NDA review but ultimately decided against it, citing concerns about study integrity and the small amount of data likely to be available.
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A Pink Sheet timeline shows the regulatory highs and lows of AMX0035's path to US approval for treatment of amyotrophic lateral sclerosis.
Launched in part to help resurrect AMX0035’s regulatory prospects, the multiregional PHOENIX study is winding down in the US. There still may be some risk of potential dropouts, however.
Office of Neuroscience Director Billy Dunn touted FDA’s ability to withdraw approval for AMX0035 if subsequent data indicate a lack of substantial evidence of effectiveness. However, this provision appears to have been infrequently used and involves a potentially time-consuming hearing.