Drug Profile

Zilganersen - Ionis Pharmaceuticals

Alternative Names: ION-1166998 (free acid); ION-1166998 (sodiated); ION-373; ION1166998

Latest Information Update: 18 Mar 2026

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At a glance

Originator Ionis Pharmaceuticals
Class Antisense oligonucleotides
Mechanism of Action Glial fibrillary acidic protein expression inhibitors; RNA interference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Alexander disease
New Molecular Entity Yes

Highest Development Phases

Preregistration Alexander disease

Most Recent Events

02 Mar 2026 Ionis Pharmaceuticals has patent protection for zilganersen
25 Feb 2026 Ionis Pharmaceuticals expects approval decision from US FDA for NDA in second half of 2026
25 Feb 2026 Ionis Pharmaceuticals initiates a Expanded Access Program (EAP) for Alexander disease in USA, prior to February 2026

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