March 2, 2021
FDA knows that ALS patients, their families, and others in the ALS community have been closely watching the development of BrainStorm Cell Therapeutics, Inc.’s NurOwn therapy. From data that have been communicated to the ALS community, there was a lot of hope that this product could provide at least a modest breakthrough in the management of ALS, if not something more substantial. Although FDA generally cannot provide confidential information about unapproved products, given the tremendous public interest in this product, we have concluded that it is important to provide high-level information about the status of the NurOwn development program.
With the recent completion of a randomized phase 3 controlled clinical trial comparing NurOwn to placebo, it has become clear that data do not support the proposed clinical benefit of this therapy. Data indicated that none of the primary or secondary endpoints were met in the group of patients who were randomized. For the main (primary) endpoint, 27.7% of people given the placebo were scored as responding compared to 32.6% of people given NurOwn. The 4.9% absolute difference in responders was not at all statistically significant, and the small difference between the two groups was most likely due to chance. In addition, there was a modest excess in deaths in those treated with NurOwn, the significance of which is unclear at this time. If BrainStorm plans further studies of NurOwn to determine if the product can provide clinical benefit to individuals with ALS, FDA will continue to provide advice to the company on their development program.
FDA continues to recognize the critical unmet medical need for new, effective treatments for ALS. We are committed to working with sponsors of novel therapies and the ALS community to facilitate development and approval of agents to treat this devastating disease. We are prepared to use all expedited development and approval pathways available to us to further this mutual goal.