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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: 07 Oct 2024

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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

  • 01 Oct 2024 Zilganersen - Ionis Pharmaceuticals receives Fast Track designation for Alexander-disease [Intrathecal,Injection] (In adolescents, In children, In the elderly, In infants, In adults) in USA
  • 22 Apr 2022 Phase-II/III clinical trials in Alexander-disease (In children, In adults, In infants, In the elderly, In adolescents) in Australia (Intrathecal) (NCT04849741; EudraCT2020-000976-40)
  • 22 Apr 2022 Phase-II/III clinical trials in Alexander-disease (In children, In adults, In infants, In the elderly, In adolescents) in Israel (Intrathecal) (NCT04849741; EudraCT2020-000976-40)

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