US FDA panel votes against BrainStorm's ALS therapy over effectiveness concern
By Mariam Sunny
(Reuters) -Advisers to the U.S. health regulator on Wednesday voted against BrainStorm Cell Therapeutics' therapy for a rare and fatal neurodegenerative disease called amyotrophic lateral sclerosis (ALS), marking the newest hurdle in a lengthy regulatory review.
While 17 panel members voted that the data presented does not demonstrate substantial evidence of the effectiveness of NurOwn for the treatment of mild-to-moderate ALS, one voted in favor and one member abstained.
The panel's decision follows years of back-and-forth in communication between the agency and the company on the clinical benefit of NurOwn after the therapy failed to meet the main goal in a late-stage study in 2020.
"As a person living with ALS... I am very sensitive to approving a drug... that could ultimately result in more harm than good in the long run," said Andrew Buckley, one of the voting members of the panel.
Brainstorm has said data from new analyses showed that the therapy was beneficial in treating a smaller subset of patients with ALS and reduced markers of nerve degeneration.
The company will explore all options available in the coming weeks, BrainStorm co-CEO Stacy Lindborg said in an emailed statement.
"We firmly believe that the data for NurOwn presented today provide a compelling case for approval, with clinical evidence in those with less advanced disease supported by strong and consistent biomarker data that are predictive of clinical response," Lindborg said.
The U.S. Food and Drug Administration's (FDA) staff reviewers said on Monday there is not enough evidence to support NurOwn's effectiveness and that there are large amounts of missing data in the company's application.
"Providing false hope can be ethically problematic and false hope is provided when the probability of a positive outcome is overestimated. And I think that seems to be the case here," said Lisa Lee, one of the panelists.
BrainStorm Cell earlier this year requested the FDA to consider the application for a smaller group of patients with mild-to-moderate ALS, instead of all patients, after the agency refused to accept the filing based on the failed trial.
NurOwn cell therapy uses stem cells harvested from each patient's bone marrow that are then manipulated to secrete high levels of certain proteins, which promote the growth of nerve cells.
The FDA has given traditional approval for three drugs - Japanese firm Mitsubishi Tanabe's Radicava, generic drug Riluzole, and Amylyx Pharmaceuticals' drug Relyvrio - for the treatment of ALS to date.
Biogen's Qalsody received the agency's accelerated approval in April, with traditional approval pending for data to present the drug's effectiveness.
The FDA generally follows the advice of its panel, but is not bound to do so. The agency is expected to make a decision on the drug by Dec. 8.
(Reporting by Mariam Sunny in Bengaluru; additional reporting by Shubhendu Deshmukh; Editing by Shinjini Ganguli, Shounak Dasgupta and Shweta Agarwal)
