BrainStorm reports FDA committee votes that NurOwn is not effective for ALS

BrainStorm announced the outcome of the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee meeting to review the biologics license application, or BLA, for NurOwn, an investigational mesenchymal stem cell therapy for the treatment of amyotrophic lateral sclerosis, or ALS. The Committee voted that NurOwn did not demonstrate substantial evidence of effectiveness for treatment of mild to moderate ALS. “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,” said Stacy Lindborg, PhD, co-CEO of BrainStorm. “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. We truly did our best to make the NurOwn data clear to the FDA Advisory Committee. Unfortunately, had more time and opportunity been allowed, many remaining questions posed by Advisory Committee members could have been sufficiently addressed.”