FDA staff reviewers raise concerns over BrainStorm Cell’s ALS therapy

FDA staff reviewers raise concerns over BrainStorm Cell’s ALS therapy

Sept 25 (Reuters) – The U.S. Food and Drug Administration’s staff reviewers raised concerns on Monday over the safety and efficacy of BrainStorm Cell Therapeutics’ (BCLI.O) amyotrophic lateral sclerosis (ALS) therapy, sending the company’s shares down 50% in premarket trading.

BrainStorm therapy NurOwn is being reviewed for the treatment of ALS, also known as Lou Gehrig’s Disease, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.

The FDA, in its briefing documents, said it does not believe there is sufficient evidence to support NurOwn’s clinical benefit, adding there were large amounts of missing data.

The health regulator also identified a higher incidence of deaths in the treatment group and said it indicates a lack of survival benefit and warrants further investigation.

A panel of external advisors to the FDA is set to vote on the effectiveness of the therapy in ALS patients on Wednesday.