Quick answers
- What is Onasemnogene abeparvovec-brve used for?
- The FDA-approved label (Itvisma, Novartis Innovative Technologies Inc., June 16, 2026) says: “ITVISMA is indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in survival motor neuron 1 (SMN1) gene. ITVISMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in SMN1 gene.” This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- What are the side effects of Onasemnogene abeparvovec-brve?
- The “Adverse Reactions” section of the FDA label (Itvisma, Novartis Innovative Technologies Inc., June 16, 2026) begins: “The most common adverse reactions that occurred in at least 10% of patients were upper respiratory tract infection, upper gastrointestinal symptoms, pyrexia, and headache.” … This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- What are the drug interactions of Onasemnogene abeparvovec-brve?
- The “Drug Interactions” section of the FDA label (Itvisma, Novartis Innovative Technologies Inc., June 16, 2026) begins: “Adjust patient’s vaccination schedule to accommodate concomitant corticosteroid administration prior to and following ITVISMA injection [see Dosage and Administration (2.1)]. Certain vaccines, such as measles, mumps, and rubella (MMR) and varicella, are contraindicated for patients on a substantially immunosuppressive steroid dose (i.e., ≥ 2 weeks of daily receipt of 20 mg or 2 mg/kg body weight of prednisone or equivalent).” … This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- Does Onasemnogene abeparvovec-brve have a boxed warning?
- Yes. The FDA label for Itvisma has a boxed warning (“WARNING: SERIOUS LIVER INJURY”), the FDA’s strongest warning. Read it on the full label and ask your pharmacist.
- Is there a shortage of Onasemnogene abeparvovec-brve?
- No current shortage is listed by the FDA for the products of this medicine (FDA data from October 30, 2026).
- Has Onasemnogene abeparvovec-brve been recalled?
- No recall linked to the products of this medicine appears in the FDA enforcement reports we use.
- Who makes Onasemnogene abeparvovec-brve?
- 1 company lists prescription products of Onasemnogene abeparvovec-brve in the FDA NDC Directory, including Novartis Innovative Technologies Inc.
What Onasemnogene abeparvovec-brve is used for
Quoted from the “Indications and Usage” section of the FDA-approved label.
ITVISMA is indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in survival motor neuron 1 (SMN1) gene. ITVISMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in SMN1 gene.
— FDA label: Itvisma, Novartis Innovative Technologies Inc., effective June 16, 2026. Read the full label on DailyMed →
Label quoted: the one for the most common form (injection, suspension, intrathecal), Itvisma, Novartis Innovative Technologies Inc.; it covers 1 product in the FDA NDC Directory. The side effects, interactions, dosage and pregnancy quotes below come from this same label.
This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
Boxed warning
A boxed warning (“black box”) is the FDA’s strongest warning on a drug label, for serious or life-threatening risks.
This label has a boxed warning. Its heading: WARNING: SERIOUS LIVER INJURY
. Read it on the full label →
Onasemnogene abeparvovec-brve boxed warning: what the label says
Quoted from the boxed warning of the FDA-approved label.
WARNING: SERIOUS LIVER INJURY Acute serious liver injury and elevated aminotransferases can occur with ITVISMA. [see Warnings and Precautions (5.1)] Patients with preexisting liver impairment may be at higher risk. [see Warnings and Precautions (5.1)] Prior to intrathecal injection, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroid before and after ITVISMA injection.
Read more of this section
Continue to monitor liver function for at least 3 months after injection, and at other times as clinically indicated. [see Dosage and Administration (2.1, 2.4)]. WARNING: SERIOUS LIVER INJURY See full prescribing information for complete boxed warning. Acute serious liver injury and elevated aminotransferases can occur with ITVISMA. (5.1) Patients with preexisting liver impairment may be at higher risk. (5.1) Prior to intrathecal injection, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroid before and after ITVISMA injection. Continue to monitor liver function for at least 3 months after injection, and at other times as clinically indicated. (2.1, 2.4)
— FDA label: Itvisma, Novartis Innovative Technologies Inc., effective June 16, 2026. Read the full label on DailyMed →
Onasemnogene abeparvovec-brve side effects
Quoted from the “Adverse Reactions” section of the FDA-approved label. Tell your doctor about any side effect that worries you.
The most common adverse reactions that occurred in at least 10% of patients were upper respiratory tract infection, upper gastrointestinal symptoms, pyrexia, and headache.
Read more of this section
(6) To report SUSPECTED ADVERSE REACTIONS, contact Novartis Gene Therapies at 1-833-828-3947 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 product and may not reflect the rates observed in practice. The safety data described in this section reflects exposure of ITVISMA in two clinical studies, Study 1, a randomized, sham-controlled study which evaluated the safety of ITVISMA in 126 patients with spinal muscular atrophy (SMA) and Study 2, an open-label-single arm study which evaluated safety of ITVISMA in 27 patients with SMA who were previously treated with nusinersen (at least 4 months washout) or risdiplam (at least 15 days washout). In Study 1, a total of 75 patients received a single intrathecal injection of ITVISMA at a fixed dose of 1.2 x 10 14 vg and 51 patients underwent a sham-procedure [see Clinical Studies (14)]. In Study 2, a total of 27 patients received a single intrathecal injection of ITVISMA at a fixed dose of 1.2 x 10 14 vg. The patients were followed for a duration of 52 weeks for both studies. In Study 1, serious adverse reactions were reported in four patients (5%) including elevated liver enzymes (n=1), sensory disturbance (n=2), and vomiting (n=1). The most frequent adverse reactions occurring in ≥ 2% of patients in Study 1 are summarized in Table 3 below. Table 3: Adverse Reactions Occurring in ≥2% of Patients or with higher frequency in ITVISMA-treated Patients compared to Sham group in Study 1 Adverse reactions ITVISMA Sham (N = 75), n (%) (N = 51), n (%) * Is a composite that includes multiple related terms a) Two patients had ALT elevations of 20 times the upper limit of normal (ULN). b) Signs and symptoms that may be suggestive of dorsal root ganglion (DRG) toxicity occurred within 3 weeks of ITVISMA injection and stabilized but remained unresolved at the end of study period. c) Occurred 154 days after the sham procedure and resolved after 15 days without intervention.
— FDA label: Itvisma, Novartis Innovative Technologies Inc., effective June 16, 2026. This is part of the section. Read the full label on DailyMed →
Onasemnogene abeparvovec-brve drug interactions
Quoted from the “Drug Interactions” section of the FDA-approved label. Tell your pharmacist about every medicine, vitamin and herbal product you take.
Adjust patient’s vaccination schedule to accommodate concomitant corticosteroid administration prior to and following ITVISMA injection [see Dosage and Administration (2.1)]. Certain vaccines, such as measles, mumps, and rubella (MMR) and varicella, are contraindicated for patients on a substantially immunosuppressive steroid dose (i.e., ≥ 2 weeks of daily receipt of 20 mg or 2 mg/kg body weight of prednisone or equivalent).
Read more of this section
Adjust patient’s vaccination schedule to accommodate concomitant corticosteroid administration prior to and following ITVISMA injection. (7)
— FDA label: Itvisma, Novartis Innovative Technologies Inc., effective June 16, 2026. Read the full label on DailyMed →
Onasemnogene abeparvovec-brve dosage
Quoted from the “Dosage and Administration” section of the FDA-approved label. Your own dose is the one your doctor prescribes: do not change it on your own.
For single-dose intrathecal injection only. (2) The recommended dose of ITVISMA is 1.2 × 10 14 vector genomes (vg). (2.2) Administer ITVISMA as an intrathecal bolus injection over approximately 1 to 2 minutes. (2.4) Postpone ITVISMA in patients with infections until the infection has resolved and the patient is clinically stable.
Read more of this section
(2.1) Starting one day prior to ITVISMA injection, administer systemic corticosteroids equivalent to oral prednisolone at 1 mg/kg of body weight per day for a total of 30 days. At the end of the 30-day period, check liver function by clinical examination and by laboratory testing. For patients with unremarkable findings, taper the corticosteroid dose gradually over the next 28 days. If liver function abnormalities persist, continue systemic corticosteroids (equivalent to oral prednisolone at 1 mg/kg/day) until findings become unremarkable, and then taper the corticosteroid dose gradually over the next 28 days or longer if needed. Do not stop systemic corticosteroids abruptly. (2.2) If at any time patients do not respond adequately to the equivalent of 1 mg/kg/day oral prednisolone, based on the patient’s clinical course, prompt consultation with a gastroenterologist or hepatologist and adjustment to the recommended corticosteroid regimen may be considered. (2.2) 2.1 Critical Dosing Information For single-dose intrathecal injection only. Patients previously treated with ZOLGENSMA (onasemnogene abeparvovec-xioi) should not be treated with ITVISMA [see Clinical Pharmacology (12.1)]. ITVISMA should only be administered intrathecally using a lumbar puncture by healthcare professionals (e.g., interventional radiologist or neurologist) experienced in performing lumbar punctures. Prior to ITVISMA injection: Due to the increased risk of serious systemic immune response, administer ITVISMA to patients who are clinically stable in their overall baseline health status (e.g., hydration and nutritional status, absence of infection, respiratory status) prior to administration. Postpone ITVISMA in patients with active or recent infections, until the infection has resolved, and the patient is clinically stable. Clinical signs or symptoms of infection should not be evident at the time of ITVISMA injection. Assess vaccination status. Vaccination status should be up-to-date prior to ITVISMA administration. Recommend seasonal prophylaxis against respiratory syncytial virus (RSV). Assess liver function (clinical examination and laboratory testing including aspartate aminotransferase (AST), alanine aminotransferase (ALT), albumin, prothrombin time, partial thromboplastin time (PTT), international normalized ratio (INR), and total bilirubin) [see Warnings and Precautions (5.1), Use in Specific Populations (8.6)]. Obtain creatinine and complete blood count (including hemoglobin and platelet count) [see Warnings and Precautions (5.2, 5.4)]. Perform baseline testing for the presence of anti-AAV9 antibodies.
Table from the label
| Pre-Injection | - |
|---|---|
| 24 hours prior to ITVISMA injection | Oral prednisolone 1 mg/kg/day (or equivalent) |
| Post-Injection | - |
| 30 days (including the day of ITVISMA administration) | Oral prednisolone 1 mg/kg/day (or equivalent) |
| Followed by 28 days: For patients with unremarkable findings (normal clinical exam, total bilirubin, and ALT and AST levels below 2 × ULN) or For patients with liver function abnormalities at the end of the 30 day period: continue until the AST and ALT values are both below 2 × ULN and all other assessments return to normal range, and then taper the corticosteroid dose over the next 28 days or longer if needed. | Systemic corticosteroids should be tapered gradually Taper prednisolone (or equivalent) Systemic corticosteroids (equivalent to oral prednisolone 1 mg/kg/day) Systemic corticosteroids should be tapered gradually |
— FDA label: Itvisma, Novartis Innovative Technologies Inc., effective June 16, 2026. This is part of the section. Read the full label on DailyMed →
Onasemnogene abeparvovec-brve in pregnancy
Quoted from the pregnancy part of the FDA-approved label. Ask your doctor before you take any medicine while pregnant or planning a pregnancy.
Risk Summary There are no clinical studies in pregnant women to inform a product-associated risk. Animal reproduction studies in mice have shown no risk of fetal abnormalities nor transfer to mouse fetus with intravenous (IV) administration of onasemnogene abeparvovec on gestational day 6 at dose levels equivalent to at least 7 times the clinical intrathecal dose. It is not known whether ITVISMA has the potential to be transferred to the fetus in humans.
Read more of this section
Therefore, women who are pregnant or may become pregnant should only be treated with ITVISMA after a thorough benefit-risk evaluation. In the United States general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data In an embryo-fetal development study in mice, pregnant animals received an IV dose of either 1.1 x 10 13 vg/kg or 1.1 x 10 14 vg/kg of onasemnogene abeparvovec on gestational day 6, which resulted in no evidence of maternal toxicity, embryo-fetal toxicity, teratogenicity, or reduced viability. Onasemnogene abeparvovec DNA was detected in the placenta at low levels but was not detected in any fetal tissue on gestational day 18. The no observed adverse effect level for maternal and developmental toxicity is 1.1 x 10 14 vg/kg, corresponding to at least 7 times the recommended clinical IT dose level based on body weight.
— FDA label: Itvisma, Novartis Innovative Technologies Inc., effective June 16, 2026. Read the full label on DailyMed →
Forms and how it is given
| Dosage forms | Injection, suspension |
|---|---|
| Route | Intrathecal |
| Prescription products | 1 in the FDA NDC Directory |
| Brand names | Itvisma |
Companies that list this medicine
Labelers in the FDA NDC Directory: the company named on the package (maker or distributor), with the number of prescription products.
FDA approval history
- BLA: Biologics License Application: how a biologic medicine (made from living cells) is approved.
| Application | Sponsor | Approved | Later changes approved |
|---|---|---|---|
| BLA125856 | — | Date not on file | — |
Shortage status
The FDA drug shortage list does not show these products.
Recalls
No recall linked to these products (by their NDC code) appears in the FDA enforcement reports.
Official identifiers
| RxCUI (RxNorm) | 2727307, 2727314 |
|---|---|
| SPL set ID of the quoted label | ab2e1c6c-4f95-4243-9296-1734e0a30591 |
| FDA labels (SPL) | 1 |
| FDA applications | BLA125856 |
| Active ingredients (FDA) | ONASEMNOGENE ABEPARVOVEC |
Next steps
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Sources
- FDA-approved label on DailyMed (National Library of Medicine) — Itvisma, Novartis Innovative Technologies Inc., effective June 16, 2026; via openFDA, FDA data exported October 30, 2026, retrieved October 31, 2026
- NDC Directory (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drugs@FDA (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drug recall enforcement reports (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 27, 2026, retrieved October 31, 2026
- Drug shortages (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
openFDA data is provided by the FDA as is; it may be incomplete. Always rely on the label that comes with your medicine. FDA Enforcement Report search →