The Food and Drug Administration has set a date for an upcoming meeting in which an outside group of experts will evaluate an experimental ALS medicine the agency has appeared wary of approving.
The medicine’s developer, Brainstorm Cell Therapeutics, disclosed Tuesday that the meeting will take place on Sept. 27. It will focus on an approval application Brainstorm recently filed, and have representatives from the company and the FDA present their cases to a committee that advises the agency on cell, tissue and gene therapies.
Brainstorm’s treatment, known as NurOwn, is made from stem cells taken from a patient’s bone marrow. The company engineers these cells so they secrete molecules that help protect and grow neurons, and then implants them back into the patient with the aim of slowing or stopping neurodegenerative diseases. NurOwn has been tested as a possible treatment for both multiple sclerosis and ALS, or amyotrophic lateral sclerosis, and Brainstorm continues to investigate its potential in other disorders like Huntington’s disease and Parkinson’s disease.
In 2016, Brainstorm said NurOwn had shown promise in a small study of about 50 ALS patients. Those results encouraged the company to push its medicine into late-stage clinical testing, and a larger, roughly 260-person trial began the following year. But by late 2020, that trial had found NurOwn to be no better than a placebo at slowing the functional decline associated with ALS.
Brainstorm executives still believed that, in spite of the failure, enough positive data had been collected to warrant an approval. However, the FDA wasn’t so convinced.
Senior leadership from the agency met with Brainstorm shortly after results from the larger study became available and, according to the company, said the evidence wasn’t substantial enough to support an approval application.
Brainstorm ultimately decided to ask for approval anyway, a move that was met with pushback from FDA staff, who late last year chose not to review the application. That decision led Brainstorm to use a rare tactic in drug regulations — a “file over protest” — which forces the FDA to review an application even if there are reservations. The agency is now set to issue an approval verdict by Dec. 8.
Before that deadline, Brainstorm will get another chance to make a case for its medicine at the upcoming advisory committee meeting.
“We remain confident in NurOwn and believe our data support regulatory approval,” Stacy Lindborg, BrainStorm’s co-CEO, said in a statement. “As is the case with most ALS research, our clinical program generated complex results, which deserve a thoughtful and holistic review by scientists, ALS experts, FDA reviewers, advocates, and patients.”
The FDA has, over the past year or so, held advisory meetings for other closely watched ALS drugs like Amylyx Pharmaceuticals’ Relyvrio and Biogen’s Qalsody. While there were limitations in the data supporting both of those drugs, they each received support from the outside experts and were later approved.
"We are encouraged by the regulatory flexibility that the FDA has shown over the last year in ALS broadly, and with respect to NurOwn in particular, and believe an advisory committee meeting is good for patients," said Chaim Lebovits, BrainStorm’s other co-CEO.
Shares of Brainstorm stock currently trade a little over $3 apiece, or more than double what they were valued at before the company filed over protest.