Zanvastro (zilganersen) is filed as antisense oligonucleotide. The sections below restate public label fields stored for this profile.
1 INDICATIONS AND USAGE 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. ( 1)
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.
5 WARNINGS AND PRECAUTIONS 5.1 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. In addition, nonserious adverse drug reactions of CSF WBC increased have also been reported with ZANVASTRO [see Adverse Reactions ( 6.1)] .
4 CONTRAINDICATIONS None. None. ( 4)
Vomiting (>25%); Back pain (>25%); Cough (>25%); Headache (>25%); Post-lumbar puncture syndrome (>25%)
6 ADVERSE REACTIONS The following clinically significant adverse reactions are described elsewhere in the labeling: Aseptic Meningitis [see Warnings and Precautions ( 5.1)] 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. ( 6.1) To report SUSPECTED ADVERSE REACTIONS, contact Ionis Pharmaceuticals, Inc. at 1-833-644-6647 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of ZANVASTRO was evaluated in 53 pediatric and adult patients with Alexander disease in Study 1 [see Clinical Studies ( 14)] . Thirty-eight patients received ZANVASTRO for 1 year, and 18 patients received ZANVASTRO for 2 years. The median exposure was 60 weeks in all groups. The most common adverse reactions during the Double-Blind Treatment Period (incidence >25% of patients treated with ZANVASTRO 50 mg and greater than control) were vomiting, back pain, cough, headache, and post-lumbar puncture syndrome. Table 3 includes the common adverse reactions that occurred in ≥10% of patients treated with ZANVASTRO and 10% greater than control. Table 3. Adverse Reactions in Patients with Alexander Disease that Occurred in ≥10% of Patients Treated with ZANVASTRO and 10% Greater than Control Adverse Reaction ZANVASTRO 50 mg (N=24) % Control (N=17) % Vomiting 50 29 Back pain 50 18 Cough 38 18 Headache 29 12 Post lumbar puncture syndrome 29 6 Arthralgia 25 6 Oropharyngeal pain 21 0 Dysphagia 17 6 Patients Less Than 2 Years of Age The adverse reactions of patients less than 2 years of age are expected to be similar to that of pediatric patients 2 years of age and older. Increased White Blood Cell Count in the CSF Pleocytosis (or increased white blood cell count in CSF) was observed in 7 (29%) patients treated with ZANVASTRO 50 mg following the administration of the first 2 to 4 doses, compared to 3 (18%) patients in the control group in the double-blind treatment period of the Main Study, and in an additional 6 patients treated with ZANVASTRO in the open-label extension period [see Warnings and Precautions ( 5.1)] .
2 DOSAGE AND ADMINISTRATION ZANVASTRO is administered intrathecally. ( 2.1) The recommended dosage is 50 mg every 3 months. ( 2.1) Dilution, Preparation, and Administration ZANVASTRO must be diluted with accompanying artificial cerebrospinal fluid (aCSF) diluent prior to administration. ( 2.2) After dilution, administer as an intrathecal bolus injection over 1 to 3 minutes. ( 2.3) See full prescribing information for complete preparation and administration instructions. ( 2.2, 2.3) 2.1 Recommended Dosage Administer ZANVASTRO intrathecally after dilution using a lumbar puncture by, or under the direction of, healthcare professionals experienced in performing lumbar punctures. The recommended dosage of ZANVASTRO is 50 mg intrathecally every 3 months. The final dose volume of the intrathecal injection is determined by the patient’s age [see Dilution and Preparation Instructions ( 2.2)] . Missed Dose(s) If a dose of ZANVASTRO is missed, administer ZANVASTRO as soon as possible. Resume treatment at the recommended dosing frequency from the date of the most recently administered dose. 2.2 Dilution and Preparation Instructions Read the entire Instructions for Use provided in the carton before diluting and preparing ZANVASTRO for intrathecal administration. Each carton contains 1 vial of ZANVASTRO and 1 vial of diluent to be used together to prepare a single dose. The diluent vial contains more volume than needed. Discard excess diluent prior to dilution as instructed below. Prepare ZANVASTRO according to the instructions below and in Table 1. Use aseptic technique when preparing and administering ZANVASTRO intrathecally. Use a 3 mL and/or 10 mL syringe with an 18G to 22G needle to withdraw excess aCSF diluent from the diluent vial as determined by Table 1 based on the patient’s age. Keep the diluent vial containing the remaining diluent for Step 3. Discard the syringe(s) containing the excess diluent. Use a 3 mL syringe with an 18G to 22G needle to withdraw 2.8 mL of ZANVASTRO from the ZANVASTRO vial. Add the withdrawn volume of ZANVASTRO to the diluent vial. Gently invert the diluent vial (which now also contains ZANVASTRO) several times to mix the solution. Attach an 18G or 19G filter needle (5 micron) to a 20 mL syringe. Withdraw the recommended dose volume of diluted ZANVASTRO from the diluent vial as determined by Table 1 based on the patient’s age. Remove the filter needle. Attach a syringe tip cap to the prepared dosing syringe. Label the prepared dosing syringe per institution procedures prior to use or transport. Discard any unused portion of the diluted solution and any unused contents of the ZANVASTRO vial. Table 1. ZANVASTRO Dilution Procedure Patient Age Volume of excess aCSF diluent to withdraw from the diluent vial and discard Volume of ZANVASTRO to add to the diluent vial Volume of diluted ZANVASTRO to withdraw from the diluent vial for administration (dose volume) Less than 2 years old 12.6 mL 2.8 mL 10 mL 2 to 7 years old (inclusive) 7.0 mL 2.8 mL 15 mL 8 years old and above 1.4 mL 2.8 mL 20 mL Storage of Prepared Dosing Syringe ZANVASTRO contains no preservatives. Once drawn into the dosing syringe, the solution must be administered within: 4 hours of preparation if kept at room temperature up to 30°C (86°F). 24 hours of preparation if refrigerated at 2°C to 8°C (36°F to 46°F). Do not freeze. Do not expose to heat. Protect the prepared dosing syringe from light as a precaution. 2.3 Administration Instructions Procedural Preparation Instructions If indicated by the clinical condition of the patient, consider sedation or local anesthesia. If indicated by the clinical condition of the patient, consider imaging to guide intrathecal administration of ZANVASTRO. Evaluate patients prior to intrathecal injection for factors that increase risks associated with lumbar puncture. Administration If the prepared dosing syringe is refrigerated prior to administration, allow the dosing syringe to warm to room temperature for at least 30 minutes prior to use for patient comfort. Do not use external heat sources to warm the dosing syringe [see Storage and Handling (16.2)] . Prior to administration, remove a volume of the patient’s CSF approximately equal to the intended dose volume as appropriate for age, based on Table 2, using a lumbar puncture needle. Table 2. Recommended Injection Dose Volume Age Injection Dose Volume After Dilution Less than 2 years old 10 mL 2 years old to 7 years old (inclusive) 15 mL 8 years old and above 20 mL Administer ZANVASTRO as an intrathecal bolus injection over 1 to 3 minutes using the lumbar puncture needle. Do not flush the needle or any attached extension catheter, if used, after administration. Discard the dosing syringe.
According to its FDA label, Zanvastro (zilganersen) is approved for: 1 INDICATIONS AND USAGE 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… For medical advice, please talk to your prescriber or pharmacist.
Zanvastro (zilganersen) belongs to the antisense oligonucleotide class of medications.
The FDA label lists Zanvastro (zilganersen) as: 3 DOSAGE FORMS AND STRENGTHS Injection: 56 mg/2.8 mL (20 mg/mL) zilganersen as a clear, colorless to slightly yellow solution in a single-dose vial Artificial cerebrospinal fluid diluent: 21 mL as a clear and colorless solution in a single-dose vial for dilution of ZANVASTRO Injection: 56 mg/2.8 mL (20 mg/mL) in a single-dose vial ( 3) Artificial cerebrospinal fluid Diluent: 21 mL solution in a single-dose vial for dilution of ZANVASTRO ( 3); intrathecal route. Available strengths and packaging vary by manufacturer.
Not yet. As of the most recent FDA data we ingest, no A-rated generic equivalent is listed for Zanvastro. Availability changes as patents expire.
Our ingest of the FDA label for Zanvastro (zilganersen) does not include a Boxed Warning at this time. See the full label on DailyMed for the current, authoritative version.
The FDA Enforcement Report does not show an active/ongoing recall for Zanvastro (zilganersen) in our latest ingest. Recall data can change daily — verify at FDA.gov.
The FDA record lists the approval date for Zanvastro (zilganersen) as 2026-09-03.
The FDA label lists Ionis Pharmaceuticals Inc. as a labeler/manufacturer of Zanvastro (zilganersen). Multiple firms may market generics of the same molecule.
The FDA label states: "Storage and Handling 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…" Follow the packaging insert for the authoritative guidance.
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