NEW YORK – BrainStorm Cell Therapeutics on Wednesday said it has withdrawn its biologics license application (BLA) for NurOwn (debamestrocel), a stem cell therapy that was under review at the US Food and Drug Administration as a treatment for amyotrophic lateral sclerosis (ALS).
The company decided to withdraw the application after discussions with the FDA. BrainStorm still has the opportunity to submit another BLA for NurOwn, a therapy in which autologous multipotent stem cells derived from bone marrow are induced to secrete high levels of neurotrophic factors that support neuronal growth and survival.
BrainStorm was seeking FDA approval for NurOwn as a treatment for mild-to-moderate ALS, arguing that by increasing neurotrophic factors, the treatment could slow disease progression. However, in September, members of the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee nearly unanimously voted that the firm hadn't provided enough evidence of NurOwn's efficacy. Further, ahead of the advisory meeting, the FDA's reviewers also expressed "major concerns" over data provided by the company in briefing documents to the advisory panel.
Now, New York-based BrainStorm said it plans to meet with the FDA to discuss a development plan for NurOwn, including developing a study protocol for a registrational Phase IIIb clinical trial. The agency has invited BrainStorm to request an expedited face-to-face meeting to discuss the path forward for NurOwn, according to the company.
"We are confident in the data supporting the value of NurOwn as an addition to the options for treating ALS, and we have every desire to bring it to the ALS community," BrainStorm President and CEO Chaim Lebovits said in a statement. "We look forward to working with the FDA to define the path forward."