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Story summary
- The U.S. Food and Drug Administration approved Novartis' gene therapy Itvisma for patients aged 2 and older with SMA and SMN1 mutation.
- Itvisma costs $2.59 million and is delivered directly to the central nervous system, unlike Zolgensma, which is given intravenously (IV).
- Clinical trials showed significant improvements in motor function.
- Thus, the approval could reduce reliance on chronic therapies for about 9,000 people in the United States living with SMA.
