Drugs / zilganersen
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | Alexander disease | 2026-07-29 | “Zilganersen Alexander disease U.S. submission Achieved” ionis.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT04849741 | Jun 2021 → Aug 2025 overdue | Alexander disease | Ionis Pharmaceuticals, Inc. | Active not recruiting | Met primary |
| Comparator or background therapy1 trial | ||||||
| — | NCT07487389 Background | — | Alexander disease | Ionis Pharmaceuticals, Inc. | Available | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2026-09-03 | Ionis Pharmaceuticals | Regulatory ZANVASTRO™ (zilganersen) approved by the FDA as the first and only disease modifying treatment for Alexander disease (AxD) in pediatric and adult patients ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has approved ZANVASTRO™ (zilganersen) for the treatment of Alexander disease (AxD) in pediatric and adult patients. |
| 2026-04-29 | Ionis Pharmaceuticals | Regulatory Ionis reports first quarter 2026 financial results and highlights progress on key programs ionis.com ↗
Zilganersen NDA for Alexander disease accepted for Priority Review, paving way for Ionis’ first independent launch from leading neurology pipeline |
| 2026-04-21 | Ionis Pharmaceuticals | Results Ionis presents new data from pivotal study of zilganersen in Alexander disease (AxD) at AAN 2026 Annual Meeting ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced additional positive results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. |
| 2026-03-23 | Ionis Pharmaceuticals | Regulatory Ionis announces zilganersen New Drug Application for Alexander disease (AxD) accepted by FDA for Priority Review ionis.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for zilganersen, an investigational RNA-targeted medicine for Alexander disease (AxD), a rare, progressive and often fatal neurological condition. |
| 2025-12-02 | Ionis Pharmaceuticals | Regulatory Ionis receives U.S. FDA Breakthrough Therapy designation for zilganersen for Alexander disease (AxD) ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to zilganersen for the treatment of Alexander disease (AxD), a rare, progressive and often fatal neurological condition. |
| 2025-10-07 | Ionis Pharmaceuticals | Results Ionis showcases transformational medicines and industry-leading pipeline at Innovation Day ionis.com ↗
Ionis will share additional details today about the positive topline results from the pivotal study of zilganersen for Alexander disease, a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. |
| 2025-09-22 | Ionis Pharmaceuticals | Results Ionis announces positive topline results from pivotal study of zilganersen in Alexander disease ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. |
| 2024-10-01 | Ionis Pharmaceuticals | Regulatory Zilganersen granted U.S. FDA Fast Track designation for people living with Alexander disease ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted zilganersen Fast Track designation for the treatment of children and adults with an ultra-rare, progressive and ultimately fatal neurological disorder known as Alexander disease (AxD). |
All press releases naming this drug 10 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | zilganersen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04849741 ↗ |
| Known as | ION-1166998 (free acid) | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095433 ↗ |
| Route | Intrathecal | “Zilganersen will be administered by intrathecal bolus (ITB) injection” NCT04849741 ↗ |