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# Brainstorm to File BLA for Lou Gehrig’s Disease
- URL: https://www.fdaweb.com/brainstorm-to-file-bla-for-lou-gehrigs-disease/
- Published: 2022-08-17T12:00:00.000Z
- Updated: 2026-09-14T17:56:11.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5152533

Brainstorm Cell Therapeutics says it will submit a BLA for it NurOwn stem cell therapy for treating amyotrophic lateral sclerosis (ALS and also known as Lou Gehrig’s Disease). Last year, FDA released an unprecedented notice ([see story](https://www.fdaweb.com/nurown-als-phase-3-trial-doesnt-support-benefit-fda/)) saying that despite high hopes in the ALS community for NurOwn, Phase 3 trial data do not support the proposed clinical benefit of the therapy. None of the trial’s primary or secondary endpoints were met, the notice said, with no statistically significant difference between the active and placebo groups. “In addition,” it said, “there was a modest excess in deaths in those treated with NurOwn, the significance of which is unclear at this time.”

Brainstorm is dismissing FDA’s analysis and now says new analyses of the Phase 3 data have “uncovered key insights that furthered our understanding of the product mechanism of action and therapeutic potential and strengthened the conclusions of NurOwn’s efficacy…We intend to provide additional updates upon learning whether the FDA files our BLA submission.”

Specifically, Brainstorm says a [correction was made to publication *Muscle and Nerve*](https://onlinelibrary.wiley.com/doi/epdf/10.1002/mus.27697?ref=fdaweb.com) that described the results of NurOwn’s Phase 3 clinical trial in ALS “following new clinical analyses which strengthen the company's original conclusions from the trial… Analyses reported in the original publication utilized an efficacy model that unintentionally deviated from the trial’s pre-specified statistical analysis plan by erroneously incorporating interaction terms between the subgroup and treatment. The newly published results, which [include supporting information to the publication](https://onlinelibrary.wiley.com/action/downloadSupplement?doi=10.1002%2Fmus.27472&file=mus27472-sup-0001-Supinfo.pdf&ref=fdaweb.com), employ the efficacy model as pre-specified in the trial’s statistical analysis plan, correcting the analyses.”