Ionis gets Orphan Drug tag for rare neurodegenerative disease

The FDA has designated Orphan Drug status to Ionis Pharmaceuticals's (NASDAQ:IONS) ION373 for the treatment of Alexander disease, a rare neurodegenerative disease, characterized by the destruction of the myelin sheath (fatty covering that protects the nerve fiber and supports signal conduction).

Alexander disease is caused by a mutation in a protein resulting in overproduction of glial fibrillary acidic protein (GFAP) in the brain, and ION373 is antisense medicine designed to stop the mutated gene from producing excess GFAP.

https://seekingalpha.com/news/3618591-ioniss-ion373-orphan-drug-in-u-s-for-rare-neurodegenerative-disease