BrainStorm Cell Therapeutics Inc. (BCLI) announced late Wednesday the U.S. Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee voted that NurOwn did not demonstrate substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis or ALS.
The Committee met to review the Biologics License Application or BLA for NurOwn, an investigational mesenchymal stem cell therapy for the treatment of ALS.
The developer of adult stem cell therapeutics for neurodegenerative diseases noted that the Phase 3 pivotal trial of NurOwn did not reach statistical significance on the primary or secondary endpoints, likely due to a “floor effect,” which confounds measurement of disease progression in patients with more advanced disease.
Stacy Lindborg, co-CEO of BrainStorm, said, “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. 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.”
The company previously announced the FDA’s intention to hold an ADCOM meeting to review NurOwn for the treatment of ALS. BrainStorm filed a BLA for NurOwn on September 9, 2022, and received a Refusal to File or RTF letter from FDA on November 8, 2022.
The BLA was filed over protest, allowing completion of the regulatory process in the shortest time possible, and active review resumed on February 7, 2023.
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