US FDA panel votes against BrainStorm's ALS therapy (updated)

Brainstorm Cell Therapeutics

27/09/2023 23:29

(Adds details in paragraph 2, background throughout)
       Sept 27 (Reuters) - The U.S. health regulator's advisers
on Wednesday voted against BrainStorm Cell Therapeutics'
 BCLI.O  therapy designed to treat patients with amyotrophic
lateral sclerosis (ALS), a potentially fatal neurodegenerative
disease.
    While 17 panel members voted that the data presented does
not demonstrate substantial evidence of the effectiveness of the
therapy, NurOwn, for the treatment of mild-to-moderate ALS, 1
voted for its use and 1 panel member abstained.
    The panel's decision follows years of back-and-forth 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.  
    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 U.S. Food and Drug Administration's (FDA) staff
reviewers also said on Monday there is not enough evidence to
support NurOwn's effectiveness, that there are large amounts of
missing data in the company's application, and pointed to
substantial concerns over the product's manufacturing.
    The FDA generally follows the advise of its panel, but is
not bound to do so. The agency is expected to make a decision on
the drug by Dec. 8. 
    ALS, also known as Lou Gehrig's disease, is a progressive
neurodegenerative disease that affects nerve cells in the brain
and spinal cord.
    ALS affects about 20,000 U.S. patients, and most patients
die within 3 to 5 years after the onset of symptoms, according
to government data.


 (Reporting by Mariam Sunny in Bengaluru; Editing by Shinjini
Ganguli, Shounak Dasgupta and Shweta Agarwal)
 ((Mariam.ESunny@thomsonreuters.com))