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.
The ALSFRS-R 'floor effect' will be a topic of debate at the 27 September advisory committee review of NurOwn. • Source: Shutterstock
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.
In a briefing document released ahead of a 27 September meeting of the Cellular, Tissue and Gene Therapies Advisory...
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A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
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Proposal to have panelists rate the strength of efficacy and safety evidence on a numeric scale also draws support in written comments on ways to optimize the advisory committee process.
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
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