Amylyx’s ALS Drug Brings Questions On Efficacy Data Robustness To US FDA Panel
Citing numerous concerns about design of the Phase II CENTAUR trial and statistical robustness of the results, agency reviewers question whether a single study provides substantial evidence of efficacy for approval of AMX0035.
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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.
Agency’s action plan for rare neurodegenerative diseases maps out over five years, but knowledge gained can inform products currently in development; new FDA grant program looks to invest in remote use of clinical outcome assessments for amyotrophic lateral sclerosis.
BrainStorm discovered a statistical error while preparing the stem cell therapy's BLA, and corrected data show a statistically significant treatment difference for a key secondary endpoint in less advanced ALS. However, the Phase III trial still failed its primary clinical efficacy endpoint.