Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time infusion into a vein. Onasemnogene abeparvovec works by providing a new copy of the gene that makes the human SMN protein.

Why is it so expensive?

The reason for it costly price is its miniscule market size in the drug manufacturing industry and its potential to save lives. Since it is used to treat SMA, a disorder which is rare and require highly specialised drug, the expertise required to make it and the research around it has taken a huge amount of time.

The treatment cost stands at a staggering $2.125 million, and that is just for one-time treatment. The Zolgensma was approved for use by the US Food and Drug Administration (FDA) in 2019. As SMA is a degenerative disorder, long-term data on the efficacy of the drug is still nowhere near complete, but early results have suggested the single-dose therapy is able to abundantly improve motor function in young children.

Uses Zolgensma in children

Zolgensma was first clinically tested a few years ago, and the first child to receive it is now five years old. Since then, quite a number of children diagnosed with SMA have received the treatment for it. And many who underwent the treatment showed improvement.

However, while there is hope to cure children of SMA, the most challenging part of the treatment would be the cost. It is next to impossible for a regular family to tr to raise millions right after getting a life changing diagnosis for the child.

Medicines for disease in the future

For what used to be an incurable disease, now parents whose child are suffering from SMA can breathe a sigh of relief with the existence of Zolgensma. The invention of this medicine is a step forward to curing or perhaps preventing diseases that have log stumped doctors and scientists.