Molecule
Onasemnogene abeparvovec is a prescription medicine 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. MedsAcross supplies Onasemnogene abeparvovec 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
From the regulator-approved product information. Each section names its source.
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.
Source: US FDA prescribing information
ITVISMA is a non-replicating recombinant AAV vector that utilizes AAV9 capsid to deliver a functional copy of human survival motor neuron 1 gene (SMN1). The transgene DNA persists largely in episomal form in the nucleus of transduced cells. Expression of the transgene is driven by a constitutive promoter (cytomegalovirus enhanced chicken-β-actin hybrid), resulting in continuous and sustained SMN expression. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. By providing an alternative source of SMN protein expression in motor neurons, it is expected to promote the survival and function of transduced motor neurons.
Source: US FDA prescribing information
For single-dose intrathecal injection only. The recommended dose of ITVISMA is 1.2 × 10 14 vector genomes (vg). Administer ITVISMA as an intrathecal bolus injection over approximately 1 to 2 minutes. Postpone ITVISMA in patients with infections until the infection has resolved and the patient is clinically stable. 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.
Source: US FDA prescribing information
None. None.
Source: US FDA prescribing information
Boxed warning (serious liver injury): Acute serious liver injury and elevated aminotransferases can occur with ITVISMA. Patients with preexisting liver impairment may be at higher risk. Prior to intrathecal injection, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroid before and after ITVISMA injection. Hepatotoxicity: Prior to ITVISMA injection, assess liver function of patients by clinical examination and laboratory testing. Continue to monitor liver function for at least 3 months after injection, and at other times as clinically indicated. Thrombocytopenia: Monitor platelet counts before ITVISMA injection, and at least weekly for the first month and as clinically indicated until platelet counts return to baseline.
Source: US FDA prescribing information
The most common adverse reactions that occurred in at least 10% of patients were upper respiratory tract infection, upper gastrointestinal symptoms, pyrexia, and headache.
Source: US FDA prescribing information
Adjust patient’s vaccination schedule to accommodate concomitant corticosteroid administration prior to and following ITVISMA injection. 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).
Source: US FDA prescribing information
Product is shipped and delivered frozen (≤ -60°C [-76°F]) in a single-dose clear vial. Upon receipt, immediately place the carton in a refrigerator at 2°C to 8°C (36°F to 46°F). ITVISMA is stable for 14 days from receipt when stored at 2°C to 8°C (36°F to 46°F).
Source: US FDA prescribing information
For healthcare professionals and buyers. This is not medical advice. Treatment decisions belong to the prescriber.
2 presentations of Onasemnogene abeparvovec, sorted by availability.
| Medicine | Manufacturer | Register | Availability |
|---|---|---|---|
| Zolgensma kit Cold chain | Novartis Innovative Technologies | US FDA | Available on request |
| Itvisma 40000000000000 units/mL injectable suspensionIntrathecal Cold chain | Novartis Innovative Technologies | US FDA | Available on request |
2 brand names across 1 markets.
Approval facts from US FDA, EMA and CDSCO data, and registration status by country where we hold register data.
Yes, on request. MedsAcross lists 2 presentations of Onasemnogene abeparvovec and sources them for licensed hospitals, importers and physicians against valid import documents.
The catalogue lists 2 brand names for Onasemnogene abeparvovec, including Itvisma, Zolgensma.
Yes. Onasemnogene abeparvovec has a central EU marketing authorisation (status: authorised), according to European Medicines Agency data.
At least one listed presentation of Onasemnogene abeparvovec must be kept cold, so it is shipped in temperature-controlled packaging. Check the storage line on each presentation.
Onasemnogene abeparvovec is classified under ATC code M09AX09 (Onasemnogene abeparvovec).
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