Ionis Pharma: FDA Grants Breakthrough Therapy Designation To Zilganersen

(RTTNews) - Ionis Pharmaceuticals (IONS) announced that the FDA has granted Breakthrough Therapy designation to zilganersen for the treatment of Alexander disease. The FDA's designation is supported by the topline results from the pivotal study of zilganersen in children and adults living with AxD.

Zilganersen is an investigational antisense oligonucleotide medicine being evaluated as a treatment for people with genetically confirmed Alexander disease. Ionis plans to submit an NDA to the U.S. FDA in first quarter 2026 and is working to initiate an Expanded Access Program in the U.S.

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