Zilganersen

Zilganersen
Clinical data
Pronunciationzil-GAN-er-sen
Trade namesZanvastro
Other namesION-1166998, ION373
AHFS/Drugs.comZanvastro
License data
Routes of
administration		Intrathecal
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
ChEBI
ChEMBL

Zilganersen, sold under the brand name Zanvastro, is a medication used for the treatment of Alexander disease. It is a glial fibrillary acidic protein (GFAP)-directed antisense oligonucleotide.

The most common side effects include vomiting, back pain, cough, headache, and post-lumbar puncture syndrome.

Zilganersen was approved for medical use in the United States in September 2026.

Medical uses

Zilganersen is indicated for the treatment of Alexander disease.

Alexander disease is a rare, progressive neurological disorder caused by mutations in the gene that produces glial fibrillary acidic protein (GFAP). When this protein is abnormal, it accumulates in the brain's supportive cells, causing damage to the nervous system over time.

Side effects

The most common side effects include vomiting, back pain, cough, headache, and post-lumbar puncture syndrome.

History

The efficacy and safety of zilganersen were evaluated in a multi-center, randomized, controlled clinical study (NCT04849741) enrolling 49 pediatric and adult participants with Alexander disease two years of age and older, and in an open-label substudy of four participants less than two years of age.

Society and culture

Zilganersen was approved for medical use in the United States in September 2026. The US Food and Drug Administration (FDA) granted the application for zilganersen orphan drug, fast track, breakthrough therapy, and rare pediatric disease and priority review voucher designations. The FDA granted the approval of Zanvastro to Ionis Pharmaceuticals.

Names

Zilganersen is the international nonproprietary name.

Zilganersen is sold under the brand name Zanvastro.

References

  1. ↑ https://www.drugs.com/zanvastro.html
  2. 1 2 3 4 "Zanvastro (zilganersen) injection, for intrathecal use" (PDF). Retrieved 4 September 2026.
  3. 1 2 3 4 5 6 7 8 9 "FDA Approves First Drug to Treat Alexander Disease". U.S. Food and Drug Administration (FDA). 3 September 2026. Retrieved 4 September 2026. Public Domain This article incorporates text from this source, which is in the public domain.
  4. ↑ "Novel Drug Approvals for 2026". U.S. Food and Drug Administration (FDA). 3 September 2026. Retrieved 4 September 2026.
  5. ↑ World Health Organization (2022). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 88". WHO Drug Information. 36 (3). hdl:10665/363551.
  6. ↑ "Zanvastro (zilganersen) approved by the FDA as the first and only disease modifying treatment for Alexander disease (AxD) in pediatric and adult patients". Ionis Pharmaceuticals (Press release). 3 September 2026. Retrieved 4 September 2026.