Last reviewed 2026-04-20 · How we verify

Onasemnogene Abeparvovec-brve

Onasemnogene Abeparvovec-brve is a Gene Therapy Small molecule drug developed by Novartis Pharmaceuticals. It is currently FDA-approved for Spinal Muscular Atrophy (SMA). Also known as: (ITVISMA®).

ITVISMA delivers a functional SMN1 gene using an AAV9 vector to promote SMN protein expression in motor neurons.

Onasemnogene Abeparvovec-brve, marketed by Novartis Pharmaceuticals, is a gene therapy for Spinal Muscular Atrophy (SMA) that delivers a functional SMN1 gene using an AAV9 vector. Its key strength lies in providing a one-time treatment that promotes sustained SMN protein expression in motor neurons, distinguishing it from chronic therapies like Nusinersen and Risdiplam. The primary risk is the key composition patent expiry in 2028, which could lead to increased competition from generic or biosimilar products.

At a glance

Mechanism of action

ITVISMA uses a non-replicating AAV9 vector to deliver a functional copy of the SMN1 gene into cells. This gene is expressed continuously, leading to sustained production of the SMN protein, which is crucial for the survival and function of motor neurons affected by SMA.

Approved indications

• Spinal Muscular Atrophy (SMA)

Boxed warnings

• 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. 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 )

Common side effects

• Upper respiratory tract infection
• Pyrexia
• Upper gastrointestinal symptoms
• Hepatic enzyme increased
• Headache
• Dizziness
• Pain in extremity
• Thrombocytopenia
• Sensory disturbance

Drug interactions

• Measles, Mumps, and Rubella (MMR) vaccine
• Varicella vaccine

Key clinical trials

• ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM) (PHASE4)

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Onasemnogene Abeparvovec-brve CI brief — competitive landscape report
• Onasemnogene Abeparvovec-brve updates RSS · CI watch RSS
• Novartis Pharmaceuticals portfolio CI

Frequently asked questions about Onasemnogene Abeparvovec-brve

Related

• Drug class: All Gene Therapy drugs
• Target: All drugs targeting SMN1 gene
• Manufacturer: Novartis Pharmaceuticals — full pipeline
• Therapeutic area: All drugs in Neuroscience
• Indication: Drugs for Spinal Muscular Atrophy (SMA)
• Also known as: (ITVISMA®)
• Compare: Onasemnogene Abeparvovec-brve vs similar drugs
• Pricing: Onasemnogene Abeparvovec-brve cost, discount & access