Panel finds NurOwn data far below standards of recent ALS approvals

Apart from a single yes vote, from a consumer representative, and one abstention, the other 17 voting members of an FDA panel agreed with the agency that BrainStorm’s ALS candidate NurOwn did not show substantial evidence of efficacy in its Phase III trial. The vote suggests a rejection by FDA is virtually certain, and helps paint a clearer picture of the limits of regulatory flexibility in ALS.

As in many panel meetings for neurodegeneration therapies, including recent amyotrophic lateral sclerosis meetings, patients and their caregivers gave passionate pleas for approval. But panelists on the Cellular, Tissue, and Gene Therapies Advisory Committee agreed that data from  BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) neither met the one-positive-trial-with-confirmatory-evidence standard that led to approval of Relyvrio from Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) nor did it produce a substantial change in NfL, the biomarker recently used to grant accelerated approval of Qalsody tofersen from Biogen Inc. (NASDAQ:BIIB). ...