FDA Approves Gene Therapy for Spinal Muscular Atrophy

At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.

Ashley Yeager

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The US Food and Drug Administration has approved a new treatment for a rare childhood disorder that costs $2.125 million for single dose—the most expensive medicine on the market.

The medicine is designed to treat spinal muscular atrophy (SMA), a condition driven by defects in the SMN1 gene, which causes afflicted babies to lose muscle control. The illness affects about 400 babies in the US each year and kills those with the most common form of the disease in just a few years. The new treatment is a gene therapy that uses genetically modified viruses to deliver healthy copies of the SMN1 gene to patients’ cells so they can generate a protein that helps the babies develop normally.

In tests of the treatment, babies who received it by 6 months of age didn’t have as severe muscle problems as those who didn’t get the drug. Infants ...

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Meet the Author

Ashley Yeager
Ashley started at The Scientist in 2018. Before joining the staff, she worked as a freelance editor and writer, a writer at the Simons Foundation, and a web producer at Science News, among other positions. She holds a bachelor’s degree in journalism from the University of Tennessee, Knoxville, and a master’s degree in science writing from MIT. Ashley edits the Scientist to Watch and Profile sections of the magazine and writes news, features, and other stories for both online and print.
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