Onasemnogene abeparvovec-brve, the active ingredient and formal name for the gene therapies branded as Itvisma and Zolgensma.
These are one-time gene replacement therapies used to treat spinal muscular atrophy (SMA), a rare, severe neurodegenerative disease caused by a mutation in the SMN1 gene.
About Onasemnogene Abeparvovec
Uses a non-pathogenic adeno-associated virus (AAV9) vector to deliver a functional copy of the human SMN1 gene to motor neurons.
This enables the body to produce the essential SMN protein, preventing further motor neuron loss and muscle weakness.
Administration: The drug is administered as a single, one-time injection. Brand Names and Indications:
Zolgensma (onasemnogene abeparvovec-xioi) is administered intravenously for pediatric patients younger than 2 years of age.
Itvisma (onasemnogene abeparvovec-brve) is a new formulation administered intrathecally (into the spinal fluid) for adults and children aged 2 years and older.
A known risk is hepatotoxicity.