An FDA independent advisory panel will convene again to discuss Amylyx’s contested ALS drug, after a meeting in March left the drug with little hope for FDA approval. And for this second try, the odds still don’t look in Amylyx’s favor.
Amylyx’s new analyses were still not “sufficiently independent or persuasive” to serve as confirmatory evidence of AMX0035's effectiveness, the FDA said in its briefing document about the application.
This time, Amylyx submitted new analyses of survival data from its main study, along with biomarker results from a phase 2 of the drug in Alzheimer’s disease, prompting the reconvening of the advisory committee tomorrow.
One of the new analyses used participants' response rates to assess the treatment's effects. The agency said that it does “not appear that this data can be considered independent confirmatory evidence” as it uses the same data as the primary analysis.
The company also presented a survival prediction algorithm for the drug, created from natural history data from more than 10,000 ALS patients from 12 specialized centers across Europe. The FDA shot down this data as patients in the database were not in a clinical trial and found “a variety of concerns” about the reliability of the analysis, such as external controls and a lack of pre-specified protocol.
Finally, Amylyx tried utilizing a placebo crossover design to provide an estimate of the treatment’s survival benefit. The agency flagged it as not new data but simply a new...
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