FDA Grants Breakthrough Therapy Designation to Ionis Pharmaceuticals' Zilganersen for Alexander Disease
Ionis Pharmaceuticals Inc. has received Breakthrough Therapy designation from the FDA for zilganersen, a treatment for Alexander disease. This designation is for therapies showing substantial improvement over existing treatments. Zilganersen showed significant results in a pivotal study, including stabilization of gait speed. Ionis plans to submit a New Drug Application in early 2026 and start an Expanded Access Program in the U.S. There are no approved treatments for Alexander disease currently.
Ionis Pharmaceuticals Inc. has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for zilganersen, an investigational treatment for Alexander disease (AxD). This designation is granted to therapies that treat serious or life-threatening conditions and have shown preliminary clinical evidence of substantial improvement over existing treatments. Zilganersen demonstrated statistically significant and clinically meaningful results in a pivotal study, including stabilization of gait speed in affected patients. Ionis plans to submit a New Drug Application (NDA) to the FDA in the first quarter of 2026 and is preparing to initiate an Expanded Access Program in the U.S. There are currently no approved disease-modifying treatments for Alexander disease. Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ionis Pharmaceuticals Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20251202774475) on December 02, 2025, and is solely responsible for the information contained therein. © Copyright 2025 - Public Technologies (PUBT)
