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A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease

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Trial Profile

A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease

Status: Active, no longer recruiting
Phase of Trial: Phase II/III

Latest Information Update: 09 Mar 2026

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At a glance

  • Drugs Zilganersen (Primary)
  • Indications Alexander disease
  • Focus First in man; Registrational; Therapeutic Use
  • Sponsors Ionis Pharmaceuticals

Most Recent Events

  • 25 Feb 2026 According to an Ionis Pharmaceuticals media release, NDA approval from the USFDA is expected in 2H 2026.
  • 02 Dec 2025 According to an Ionis Pharmaceuticals media release, the company plans to submit an NDA to the U.S. FDA in Q1 2026.
  • 02 Dec 2025 According to an Ionis Pharmaceuticals media release, the company announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to zilganersen for the treatment of Alexander disease (AxD). The designation is supported by the topline results from this pivotal study.

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