FDA Finally to Discuss NurOwn MSC-NTF Cell Therapy for ALS

The company's persistence in bringing NurOwn to the market amidst negative regulatory feedback has garnered an AdComm meeting.

The FDA will hold an advisory committee meeting discussing BrainStorm Cell Therapeutics’ mesenchymal stem cell neurotrophic factor (MSC-NTF) cell therapy NurOwn for the potential treatment of amyotrophic lateral sclerosis (ALS), the company has announced.1

“The FDA provided us with more than 1 path to an Advisory Committee Meeting for NurOwn. Our goal has always been to make NurOwn available to people living with ALS as quickly as possible, therefore we chose the File Over Protest pathway since this offered the fastest path to an ADCOM and regulatory decision relative to other pathways provided by FDA,” Chaim Lebovits, president and chief executive officer, BrainStorm, said in the statement.1 “The ALS community needs additional treatment options now, and we firmly believe our data support regulatory approval of NurOwn. We are grateful to the FDA for the opportunity to have the clinical evidence supporting NurOwn reviewed.”

BrainStorm met with the FDA in a type A meeting on January 11, 2023, to discuss the refusal to file letter the company received in November 2022.2,3 BrainStorm submitted a biologics license application for NurOwn in April 2022 after trial data announced in 2021 from a phase 3 clinical trial (NCT03280056) prompted the FDA to recommend against BLA submission.4

"ALS is a horrific, neurodegenerative disease that moves at a terrifying speed, robbing people of their ability to move, speak, eat, and breathe. Securing an Advisory Committee Meeting representsan important step towards our goal of making NurOwn broadly available to ALS patients in urgent need of new, effective therapies. The meeting will provide an open forum for BrainStorm and the FDA, together with medical experts, statisticians, and the ALS community, to thoughtfully review all available evidence supporting NurOwn,” Stacy Lindborg, PhD, co-chief executive officer, BrainStorm, added to the statement.1 “We remain confident in NurOwn and we are committed to doing everything in our power to make the product available quickly to people living with ALS. We look forward to a robust scientific discussion.”

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New analyses announced in 2022 prompted BrainStorm to submit the BLA.5 While the phase 3 trial had failed to meet its primary endpoint in 2021 of responder rates on ALSFRS-R scores in all stages of disease progression, a newer erratum revealed that analyses that adhered more closely to prespecified sub-analyses showed statistically significant improvements (P = .050) of more than 2 points on ALSFRS-R scores in participants with a baseline score of at least 35.

“I want to thank the FDA and Brainstorm for having this important ADCOM. As was said in the petition submitted to the FDA and signed by over 30,000 people living with ALS, caregivers, and advocates, ‘an Advisory Committee meeting will allow for an independent review of NurOwn as well as the data on efficacy and safety,” Brian Wallach, a person living with ALS, added to the statement.1 “It is critical for all ALS treatments that they be given the chance to proceed to an ADCOM.’ The ALS community has waited years for this ADCOM. It is time to let the science have a full, fair, and transparent hearing so that we can get this treatment to people who are living with and dying from ALS as soon as possible.”

NurOwn is also being investigated in patients with multiple sclerosis in a phase 2 trial (NCT03799718), positive safety and efficacy data from which were released in October 2022.6

1. BrainStorm Cell Therapeutics announces FDA advisory committee meeting to review biologic license application for NurOwn® for the treatment of ALS. News release. BrainStorm Cell Therapeutics. March 27, 2023.
2. BrainStorm Cell Therapeutics announces Type A Meeting with FDA granted for NurOwn®. December 27, 2022. https://ir.brainstorm-cell.com/2022-12-27-BrainStorm-Cell-Therapeutics-Announces-Type-A-Meeting-with-FDA-Granted-for-NurOwn-R
3. BrainStorm Cell Therapeutics receives refusal to file letter from FDA for its new biologics license application for NurOwn for the treatment of ALS. News release. November 10, 2022. Accessed November 10, 2022. https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-receives-refusal-to-file-letter-from-fda-for-its-new-biologics-license-application-for-nurown-for-the-treatment-of-als-301673753.html
4. BrainStorm Announces High-Level FDA Feedback on NurOwn® ALS Clinical Development Program. News release. BrainStorm Cell Therapeutics. News release. February 22, 2021. https://ir.brainstorm-cell.com/2021-02-22-BrainStorm-Announces-High-Level-FDA-Feedback-on-NurOwn-R-ALS-Clinical-Development-Program
5. BrainStorm Cell Therapeutics announces second quarter 2022 financial results and provides a corporate update. News release. BrainStorm Cell Therapeutics. August 15, 2022. https://ir.brainstorm-cell.com/2022-08-15-BrainStorm-Cell-Therapeutics-Announces-Second-Quarter-2022-Financial-Results-and-Provides-a-Corporate-Update
6. Cohen JA, Lublin FD, Lock C, et al. Evaluation of neurotrophic factor secreting mesenchymal stem cells in progressive multiple sclerosis. Mult Scler. Published online September 14, 2022:135245852211221. doi:10.1177/1352458522112215

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