Agency Questions the Reliability of Clinical Data and Manufacturing Consistency Ahead of Advisory Committee Meeting
In a recent briefing document, the US Food and Drug Administration (FDA) has expressed significant reservations about NurOwn, BrainStorm Cell Therapeutics’ experimental stem cell therapy for amyotrophic lateral sclerosis (ALS). The agency’s concerns revolve around the lack of substantial clinical evidence supporting the treatment’s effectiveness and questions about BrainStorm’s manufacturing reliability.
HCN Medical Memo
For healthcare professionals closely following advancements in ALS treatment, the FDA’s reservations about NurOwn are a cautionary note. The lack of substantial clinical evidence and questions about manufacturing consistency not only put the therapy’s approval at risk but also underscore the challenges in developing effective treatments for ALS. The FDA’s skepticism could potentially influence future research directions and funding in the ALS therapeutic landscape.
- The FDA states that current clinical data do not provide substantial evidence to support NurOwn’s effectiveness in treating ALS.
- An FDA advisory committee meeting was held on September 27 to discuss the therapy’s efficacy.
- BrainStorm’s Phase 3 clinical trial failed to show significant differences in disease progression between patients treated with NurOwn and those given a placebo.
- Healthcare professionals note that the FDA’s skepticism could be a significant setback for ALS treatment advancements.
Over the course of the six-month trial, 10 patients given NurOwn died, compared to three given a placebo. However, the trial was not designed to assess survival.
- BrainStorm used the FDA’s “file over protest” procedure to seek a review of the application for NurOwn, with a final decision expected in December.
- The FDA also raised concerns about BrainStorm’s ability to consistently manufacture NurOwn, citing variability in the number of cells produced and their signaling molecule levels.
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