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Molecule

Zilganersen

Available on requestOrphanCold chain
Presentations
1
Brands
1
Manufacturers
1
Available from stock
0

Zilganersen is a prescription medicine indicated for the treatment of Alexander disease in pediatric and adult patients. It is available as kits. MedsAcross supplies Zilganersen for export from India to licensed hospitals, importers and physicians acting for named patients, with the documents each country requires.

Information from the US FDA prescribing information, retrieved 12 November 2026. Not yet medically reviewed.How we review

Key facts

US FDA first approval
3 September 2026
EU status
Not recorded
Orphan designation
Yes
WHO essential medicine
Not recorded
India (CDSCO)
Not yet confirmed against CDSCO approval lists
Dosage forms
Kit

Clinical information

From the regulator-approved product information. Each section names its source.

Uses

ZANVASTRO is indicated for the treatment of Alexander disease in pediatric and adult patients. ZANVASTRO is a glial fibrillary acidic protein (GFAP)-directed antisense oligonucleotide indicated for the treatment of Alexander disease in pediatric and adult patients.

Source: US FDA prescribing information

How it works

ZANVASTRO is an antisense oligonucleotide that causes degradation of glial fibrillary acidic protein (GFAP) pre-mRNA through binding to GFAP pre-mRNA, which results in a reduction of GFAP protein synthesis.

Source: US FDA prescribing information

Dosing overview

ZANVASTRO is administered intrathecally. The recommended dosage is 50 mg every 3 months. Dilution, Preparation, and Administration ZANVASTRO must be diluted with accompanying artificial cerebrospinal fluid (aCSF) diluent prior to administration. After dilution, administer as an intrathecal bolus injection over 1 to 3 minutes. See full prescribing information for complete preparation and administration instructions.

Source: US FDA prescribing information

Warnings and precautions

Aseptic Meningitis If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care. Adverse reactions of aseptic meningitis (also called chemical meningitis or drug-induced aseptic meningitis) were reported in patients treated with ZANVASTRO during the double-blind and open-label periods of Study 1. One patient experienced a serious adverse reaction of aseptic meningitis during the double-blind treatment period of Study 1, which reoccurred in the open-label extension period and required dose interruption and pretreatment with intravenous dexamethasone prior to subsequent administration of ZANVASTRO. Despite corticosteroid premedication, CSF white blood cell (WBC) and protein increased with continued exposure but the patient remained asymptomatic and did not require discontinuation from treatment.

Source: US FDA prescribing information

Side effects

The following clinically significant adverse reactions are described elsewhere in the labeling: Aseptic Meningitis Most common adverse reactions (incidence >25% patients treated with ZANVASTRO and greater than control) were vomiting, back pain, cough, headache, and post-lumbar puncture syndrome.

Source: US FDA prescribing information

Storage

Store the ZANVASTRO vial and diluent vial in the refrigerator between 2°C and 8°C (36°F and 46°F) in the original carton to protect from light. Once taken out of the refrigerator, the ZANVASTRO vial and diluent vial can be stored at room temperature up to 30°C (86°F) in the original carton for up to 14 days. If not used within the 14 days stored at room temperature, discard ZANVASTRO. Do not freeze. Do not freeze. Protect from light as a precaution. See Dosage and Administration for instructions on how to store ZANVASTRO diluted solution.

Source: US FDA prescribing information

For healthcare professionals and buyers. This is not medical advice. Treatment decisions belong to the prescriber.

All presentations

1 presentations of Zilganersen, sorted by availability.

MedicineManufacturerRegisterAvailability
Zanvastro kit Cold chainIonis PharmaceuticalsUS FDAAvailable on request

Brands by market

1 brand names across 1 markets.

Brand names by country

United States

Manufacturers

Regulatory status

Approval facts from US FDA, EMA and CDSCO data, and registration status by country where we hold register data.

RegisterStatusSource
US FDAFirst approved 3 September 2026Drugs@FDA
EU (EMA)Not recorded—
India (CDSCO)Not yet confirmed against CDSCO approval lists—
United StatesRegisteredUS FDA (NDC directory, openFDA)Checked 12 November 2026

Questions and answers

Is Zilganersen available for export from India?

Yes, on request. MedsAcross lists 1 presentations of Zilganersen and sources them for licensed hospitals, importers and physicians against valid import documents.

What brand names does Zilganersen have?

The catalogue lists 1 brand names for Zilganersen, including Zanvastro.

Is Zilganersen approved by the US FDA?

Yes. The US FDA first approved Zilganersen on 3 September 2026, according to Drugs@FDA.

Does Zilganersen need cold chain shipping?

At least one listed presentation of Zilganersen must be kept cold, so it is shipped in temperature-controlled packaging. Check the storage line on each presentation.

References

  1. ZANVASTRO prescribing information (Ionis Pharmaceuticals). U.S. National Library of Medicine, DailyMed. Retrieved 14 September 2026

Request Zilganersen for export supply

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