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FDA Grants Orphan Drug Status for ALS Candidate

Neuron illustration (NIH)

(National Institute on Aging, NIH)

BrainStorm Cell Therapeutics Inc. in Petach Tikvah, Israel says the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s NurOwn adult stem cell product candidate for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease. ALS is a progressive neurodegenerative disease of nerve cells in the brain and spinal cord; some 5,600 people in the U.S. are diagnosed with ALS each year.

The NurOwn technology processes adult human mesenchymal stem cells that are present in bone marrow and are capable of self-renewal as well as differentiation into many other cell types. The research behind the technology was among the first to differentiate adult bone marrow cells into cells capable of releasing neurotrophic factors, proteins important to the health of the mature nervous system.

Orphan drug designation, as granted by the Orphan Drug Act, is for a product to treat a rare disease or condition that affects fewer than 200,000 people in the U.S. Orphan drug designation qualifies the sponsor of the product for tax credits and seven years of marketing exclusivity, among other benefits.

BrainStorm Cell Therapeutics credits the research behind the NurOwn technology to studies conducted at Tel Aviv University. The company says it acquired the rights to develop and commercialize the technology through an exclusive, worldwide licensing agreement with the university’s technology transfer unit.

Read more: Trial Shows Drug Effect on ALS Blood Biomarker

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