Overview
Itvisma is approved by the U.S. Food and Drug Administration (FDA) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 years of age and older who have a confirmed mutation (variation) in the SMN1 gene. This gene mutation leads to reduced production of survival motor neuron (SMN) protein, which is essential for muscle function. Eligibility for treatment with Itvisma requires genetic confirmation of the SMN1 mutation, and a person must not have an active infection at the time of treatment. Itvisma is also known by its drug name, onasemnogene abeparvovec-brve.

Itvisma is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver a functional copy of the SMN1 gene directly into the fluid around the spinal cord. This is intended to increase SMN protein production, helping to improve or maintain muscle strength and function in individuals with SMA.

How do I take it?
Prescribing information states that Itvisma is administered as a single-dose intrathecal (injected into the spinal canal) bolus over one to two minutes. The treatment requires pretreatment with corticosteroids starting one day before the injection and continuing for at least 30 days. If liver function remains normal, the corticosteroids are then gradually reduced over 28 days. If liver function does not return to normal, corticosteroid treatment is continued until improvement. Itvisma should be administered exactly as prescribed by a healthcare provider.

Side effects
According to the FDA-approved prescribing label, common side effects of Itvisma include upper respiratory tract infection, upper gastrointestinal symptoms, fever, and headache.

Rare but serious side effects may include liver damage, thrombocytopenia (low platelet counts), peripheral sensory neuropathy (nerve damage that affects sensation), thrombotic microangiopathy (TMA, a condition that causes small blood clots in blood vessels), elevated cardiac troponin I (a marker of heart muscle damage), and a potential theoretical risk of tumor development due to integration of viral DNA into the genome.

For more information about this treatment, visit:
Itvisma (Onasemnogene Abeparvovec-Brve) Suspension, for Intrathecal Injection — Novartis Gene Therapies