In overwhelmingly concluding that efficacy has not been demonstrated for the mesenchymal stromal cell therapy, panelists went beyond just the failed Phase III trial and cited BrainStorm’s ‘statistical magic’ and failure to adequately describe the mechanism of action, as well as safety and product quality concerns.
A US Food and Drug Administration advisory committee resoundingly concluded that BrainStorm Cell Therapeutics Inc. has not proven NurOwn (debamestrocel) is effective in treating amyotrophic lateral sclerosis, with panelists going well beyond just the clinical data in identifying problems with the application.
At a 27 September meeting, the Cellular, Tissue and Gene Therapies Advisory Committee voted 17-1, with one abstention, that the...
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.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
New marketing authorization applications filed with the European Medicines Agency's human medicines committee (the CHMP) for review under the EU's centralized procedure. Updated monthly.
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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Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
