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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Highly negative FDA advisory committee review and subsequent discussions with the agency made clear the only path forward was a new study, company executives said. Troubled application shows the limits of FDA’s willingness to exercise regulatory flexibility.
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.
NurOwn Adcomm Is 20 Members Strong Despite US FDA’s ‘Struggle’ To Find Unconflicted Neurology Experts
Seven panelists are regular members of the Cellular, Tissue and Gene Therapies advisory committee, while many of the 13 temporary voting members bring neurology and neuroscience expertise. In a conflict-of-interest waiver, the agency noted a struggle to find experts in clinical neurology and neurodegenerative diseases who lack disqualifying financial interests.