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Zilganersen - Ionis Pharmaceuticals

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Drug Profile

Zilganersen - Ionis Pharmaceuticals

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

Latest Information Update: 26 Sep 2025

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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

  • Phase II/III Alexander disease

Most Recent Events

  • 22 Sep 2025 Ionis Pharmaceuticals plans to submit a New drug application (NDA) for Alexander disease to the U.S. Food and Drug Administration in Q1 2026
  • 22 Sep 2025 Efficacy and adverse events data from a phase II/III trial in Alexander disease released by Ionis Pharmaceuticals
  • 22 Sep 2025 Ionis Pharmaceuticals plans a Expanded Access Program (EAP) for Alexander disease in USA

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