BrainStorm Cell Therapeutics Inc.’s mesenchymal stem cell therapy NurOwn (debamestrocel) has failed to demonstrate substantial evidence of effectiveness for the treatment of amyotrophic lateral sclerosis, and the lack of effect over placebo cannot be explained by the sponsor’s assertion of a “floor effect” with the Phase III study’s primary clinical endpoint, the US Food and Drug Administration said.
BrainStorm’s NurOwn: Cell Therapy Lacks Substantial Evidence Of Efficacy In ALS, US FDA Says
Sponsor’s assertion of a ‘floor effect’ cannot explain the lack of difference versus placebo on clinical endpoints, and there is no evidence that assessed biomarkers are reasonably likely to predict clinical benefit, agency says. Although advisory committee will focus primarily on efficacy, FDA also cites concerns about ‘grossly deficient’ product quality information.
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