Paris, France, November 23th, 2020 – BrainEver, a biotechnology company dedicated to the research and development of innovative therapies for the treatment of neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its product BREN-02, the recombinant human homeoprotein Engrailed 1 (rhEN1), for the treatment of amyotrophic lateral sclerosis (ALS).
ALS (also known as Charcot disease) is a severe neurodegenerative disease affecting motor neurons, resulting in progressive muscle weakness leading to paralysis, with a vital prognosis of approximately 2-4 years from onset. ALS is diagnosed in approximately 16,000 people each year in Europe and North America.
“In ALS, motor neurons degenerate and die, leading to progressive muscle paralysis that includes respiratory muscles. In preclinical studies, we have observed that the intrathecal lumbar administration of rhEN1, in the early symptomatic stages, restores muscle function and prevents motor neuron death”, said Prof. Alain Prochiantz, neurobiologist at the Collège de France and co-founder of BrainEver“