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Critical Importance of FDA AdComm Meeting for ALS Therapy NurOwn

Namita Goyal MD(1)

NurOwn (BrainStorm Cell Therapeutics) is an investigational therapy made up of autologous mesenchymal stromal cells secreting neurotrophic factors cells (MSC-NTF) for the potential treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The submitted biologics license application (BLA) for the agent was based on a phase 3 study (NCT03280056) in which the agent originally failed to meet its primary end point; however, it continued to have significant effects on less severe forms of ALS. In the original analysis, 33% and 28% of patients on NurOwn and placebo respectively, met the primary end point of change in disease progression of at least 1.25 points on ALS Functional Rating Scale (ALSFRS-R).

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