Onasemnogene abeparvovec

Onasemnogene abeparvovec, sold under the brand name Zolgensma among others, is a gene therapy used to treat spinal muscular atrophy, a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It works by providing a new copy of the survival of motor neuron (SMN) gene that produces the SMN protein.

Spinal muscular atrophy stems from a mutation in the survival motor neuron 1 (SMN1) gene, causing survival of motor neuron protein deficiency vital for motor neuron survival. Onasemnogene abeparvovec, a biologic medication utilizing adeno-associated virus (AAV9) virus capsids containing an SMN1 transgene, is administered to motor neurons, boosting SMN protein levels. Common side effects include vomiting and elevated liver enzymes, while more severe reactions involve liver issues and low platelet count.

Developed by AveXis and acquired by Novartis, onasemnogene abeparvovec gained various US Food and Drug Administration designations and approvals. Controversies included data manipulation concerns and delayed reporting to regulatory agencies. Onasemnogene abeparvovec's price is high, earning it the title of the world's most expensive medication at the time of commercial approval. This has later been exceeded by other gene therapies like etranacogene dezaparvovec. Several countries such as Japan, the Netherlands, Canada, Brazil and others negotiated a lower price for onasemnogene abeparvovec for their public healthcare systems.