Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced that the FDA has granted a Fast Track designation to its investigational RNA-targeted therapy zilganersen for treating Alexander disease (AxD), an ultra-rare neurological disorder.
The Fast Track designation from the FDA facilitates rapid development and expedites the review of drug candidates to treat serious conditions for which clinical data demonstrate the potential to address unmet medical needs. The goal is to make these treatments rapidly available to patients in need.
AxD is a rare neurological disease that affects a type of cell in the brain called astrocytes, which has multiple roles in the brain to support neurons and oligodendrocytes. People living with this condition experience cognitive dysfunction and progressive neurologic deterioration, including swallowing and the ability to control muscles for large movements. Per Ionis, zilganersen is the first investigational medicine in clinical development for treating AxD. There are currently no approved therapies for AxD patients.
The FDA had previously granted Orphan Drug designation and Rare Pediatric Disease designation to zilganersen for treating AxD.
Shares of Ionis have declined 22.8% so far this year compared with the industry's 1.3% fall.
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