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FDA grants orphan drug status to homeoprotein drug for amyotrophic lateral sclerosis

BrainEver, a joint laboratory (Labcom) with Collège de France, CNRS and Inserm, , has just received approval from the US Food and Drug Administration (FDA) for the Engrailed-1 protein (BREN-02), for the treatment of amyotrophic lateral sclerosis (ALS).

Diagram: After internalization, the Engrailed-1 protein acts at different levels to protect target neurons
alain Prochiantz/BrainEver. After internalization, Engrailed-1 acts at different levels to protect target neurons.

This Labcom is headed by Alain Prochiantz, holder of the Morphogenetic Processes Chair (2007-2019) at the Collège de France, and by Ariel di Nardo for the CNRS. Alain Prochiantz is also Scientific Director of BrainEver, which specializes in the development, in psychiatry and neurology, of therapeutic proteins derived from transcription factors of the homeoprotein family.

The FDA grants orphan drug status to encourage the development of therapies to treat, prevent or diagnose diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation in the U.S. recognizes the therapeutic potential of Engrailed-1, and will enable BrainEver to benefit from a number of measures and advantages, including a seven-year period of marketing exclusivity if the protein is approved for the treatment of ALS.