Dive Brief:

  • Brainstorm Cell Therapeutics said Thursday that the Food and Drug Administration has decided not to review an approval application for a cell therapy developed by the New York-based biotechnology company to treat amyotrophic lateral sclerosis, or ALS.


  • Brainstorm did not indicate why the FDA issued a so-called refuse to file letter, but said the agency indicated that a meeting can be requested to talk about the letter’s contents. Brainstorm said it will discuss its corporate strategy and plans to advance the cell therapy’s development on Monday, during a third quarter earnings call with investors.


  • Two years ago, Brainstorm’s “NurOwn,” a type of engineered stem cell therapy, failed to meet the main goal of a key clinical study evaluating ALS patients. Yet, the company continued to pursue approval, arguing there were other, positive findings from the study, in addition to the discovery of an error in the statistical methods used to assess certain results.

Dive Insight:

The FDA’s choice marks the latest setback for the NurOwn program.

When that key, late-stage clinical trial failed in late 2020, Brainstorm’s CEO Chaim Lebovits said the company was confident there would be a path forward for its medicine. Brainstorm highlighted, for example, how the trial’s placebo group performed better than expected, and how patients with early ALS appeared to have greater responses to NurOwn treatment.

But those details weren’t so convincing to regulators. By early 2021, Brainstorm had met with senior leaders at the FDA, who, according to the company, said the evidence generated by that point wasn’t substantial enough to support an approval application.

Brainstorm ultimately decided to file for approval anyway, a decision based, in large part, on a “correction” to the initial results. The company said additional analyses found an error in the statistical methods used to evaluate NurOwn on one of the trial’s secondary goals. Brainstorm said that, when the error was corrected for, its therapy performed much better on that outcome.

“While we are disappointed that the FDA has not accepted our [approval application] for NurOwn in ALS, we remain committed to NurOwn’s advancement as a treatment for this devastating disease,” Lebovits said in a statement Thursday, adding that Brainstorm intends to request a meeting with the agency to further discuss its therapy.

The FDA has shown flexibility toward ALS drugs in the past. Less than two months ago, the agency approved Amylyx Pharmaceuticals’ Relyvrio based on data from a mid-stage study of about 140 patients. According to Amylyx, the FDA originally wanted the company to run another, larger trial before filing Relyvrio for approval. But after intense pushback from patient advocacy groups, the agency agreed to review the drug while its developer ran the bigger trial.

Results from Amylyx’s smaller study showed its drug offered modest benefits in function and survival. Relyvrio is the first ALS drug to be approved in five years, joining a short list of other available medicines.

ALS is neurodegenerative disorder in which patients gradually lose their ability to perform essential functions like talking, eating and breathing. The disease is often rapid, with patients usually living between two to five years after they’re diagnosed.