FDA panel denies biologic license application for ALS stem cell therapy

The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee has voted to deny a biologics license application for NurOwn, an investigational mesenchymal stem cell therapy for the treatment of amyotrophic lateral sclerosis.

The committee voted Sept. 27 by a 17-1 margin, with one abstention, that NurOwn did not meet the criteria of whether NurOwn (debamestrocel, BrainStorm Cell Therapeutics) demonstrated substantial evidence of effectiveness for the treatment of mild to moderate ALS.

According to a release issued by BrainStorm, its phase 3 trial of NurOwn did not reach statistical significance on either primary or secondary endpoints, likely due to a “floor effect,” which confounds measurement of disease progression in patients with more advanced disease.

Analysis of the study data showed evidence of clinically meaningful efficacy in those with ALS who have not reached advanced levels of disease progression. In a pre-specified group of participants with an ALS Functional Rating Scale-Revised (ALSFRS-R) score of 35, a larger treatment effect across all endpoints with NurOwn was shown compared with placebo, which aligned with previous trials and resulted in a statistically significant difference on a key endpoint of change from baseline in ALSFRS-R, the company said in the release.

“The committee’s vote was a sad outcome for the ALS community, who have too few options to help manage this merciless and deadly disease,” BrainStorm co-CEO Stacy Lindborg, PhD, said in the release. “We firmly believe that the totality of data presented for NurOwn today provide a compelling case for approval, with clinical evidence in those with less advanced disease supported by strong and consistent biomarker data that are predictive of clinical response.”