FDA fast tracks ALS drug

October 11, 2014

FDA has designated new ALS stem cell therapy from BrainStorm Cell Therapeutics as a “fast-track” product.

FDA has designated new ALS stem cell therapy from BrainStorm Cell Therapeutics as a fast-track product.

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Amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig’s Disease, is a neurodegenerative disease that affects the nerves in the brain and spinal cord which leads to progressive paralysis throughout the body. ALS is the most common form of motor neuron disorder affecting adults in the United States and there are approximately 5,000 newly diagnosed cases every year. Currently, treatment for ALS is almost entirely supportive including pain management and speech pathology as well as nutritional support.

Israel’s Brainstorm Cell Therapeutics has developed a new treatment, called NurOwn, which is an adult stem cell therapy used to differentiate bone marrow-derived stem cells into specialized neuronal cells. NurOwn uses the patient’s own stem cells so that there is no risk of rejection and no need to treat with immunosuppressive agents that can cause serious, long-term side effects.

The FDA fast track designation is designed to facilitate and accelerate the review of drugs intended to treat serious or life-threatening conditions that show the potential to address an unmet medical need.  Drug companies must submit a request for the designation with supporting documentation for the proposed use and FDA will decide within 60 days whether the conditions for fast track have been met.

“What is so valuable about fast track designation to a small company like BrainStorm is the opportunity to have increased meetings with and more frequent written communication from the FDA," BrainStorm Chief Executive Tony Fiorino said, adding that only a small number of cellular therapies have received FDA approval.

BrainStorm’s therapy is currently being studied in a mid-stage clinical trial in patients with ALS to evaluate the safety and efficacy of therapy. The estimated completion date in the United States is December 2015.

Erin Bastick is a PharmD Candidate 2016, Ohio Northern University, Ada, Ohio, and an inpatient intern at University Hospitals, Cleveland.

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