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Zilganersen - Ionis Pharmaceuticals/Recordati

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Drug Profile

Zilganersen - Ionis Pharmaceuticals/Recordati

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

Latest Information Update: 07 Sep 2026

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At a glance

  • Originator Ionis Pharmaceuticals
  • Class Antisense oligonucleotides
  • Mechanism of Action Gene silencing; Glial fibrillary acidic protein expression inhibitors
  • 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

  • Registered Alexander disease

Most Recent Events

  • 03 Sep 2026 Registered for Alexander-disease (In adolescents, In adults, In children, In infants) in USA (Intrathecal) - First global approval (9474047)
  • 03 Sep 2026 Ionis Pharmaceuticals announces intention to submit regulatory application in Europe for Alexander disease in 2027
  • 03 Sep 2026 Ionis Pharmaceuticals announces intention to submit regulatory application in Japan for Alexander disease in 2027

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