ZANVASTRO™ (zilganersen) approved by the FDA as the first and only disease modifying treatment for Alexander disease (AxD) in pediatric and adult patients

- Provides a clinically meaningful impact on motor function and a favorable impact on other symptoms seen in AxD, an ultra-rare, progressive and often fatal neurological disorder - - Priority Review Voucher (PRV) awarded in conjunction with approval - - ZANVASTRO marks Ionis’ first independent launch from its industry-leading neurology pipeline and second independent launch this year - – Ionis to host webcast on Friday, Sept. 4 at 10:00 a.m. ET – CARLSBAD, Calif.--(BUSINESS WIRE)--Sep. 3, 2026-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has approved ZANVASTRO™ (zilganersen) for the treatment of Alexander disease (AxD) in pediatric and adult patients. ZANVASTRO is the first and only disease modifying treatment for AxD, an ultra-rare, progressive and often fatal neurological disorder that can affect motor, cognitive, autonomic and gastrointestinal function. Until now, treatment of AxD has primarily been limited to managing symptoms. ZANVASTRO is an RNA-targeted medicine designed to address the underlying disease mechanism of AxD by reducing the production of glial fibrillary acidic protein (GFAP). ZANVASTRO 50 mg is administered quarterly as an intrathecal (IT) injection. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260903826844/en/

The release as fetched from its publisher. ionis.com ↗