The FDA granted AB Science, a French company headquartered in Paris Orphan Drug Designation for masitinib for the treatment of amyotrophic lateral sclerosis (ALS). ALS is a devastating rare disease characterized by the death of neurons.  The cause is unknown in most of the cases.  There is no effective treatment and ALS is managed by relieving symptoms and extending life expectancy.  Masitinib is an oral tyrosine kinase inhibitor that targets certain types of immune cells.  It is currently in phase III clinical trials for various tumors and for inflammation related diseases including asthma, rheumatoid arthritis, Alzheimer’s disease, progressive forms of multiple sclerosis, and ALS.

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