Novartis AG announced that the U.S. Food and Drug Administration approved its gene‑replacement therapy Itvisma (onasemnogene abeparvovec‑brve) on Monday, November 24, 2025. The approval covers children two years and older, teens, and adults with spinal muscular atrophy (SMA) who have a confirmed SMN1 gene mutation, expanding the age range beyond the infant‑only indication of the company’s earlier therapy, Zolgensma.
Itvisma delivers a single intrathecal dose that replaces the missing SMN1 gene, offering a one‑time treatment that could eliminate the need for chronic, lifelong therapies currently required for SMA. The approval was based on data from the Phase III STEER study and the Phase IIIb STRENGTH study, which demonstrated statistically significant improvements in motor function and stabilization of motor abilities across the expanded patient population.
The therapy carries a wholesale acquisition cost of $2.59 million, roughly 35‑46 % less than the cumulative cost of existing chronically dosed treatments over a ten‑year horizon. Novartis has also launched a patient support program that may allow eligible patients to pay as little as $0, improving access and potentially accelerating uptake.
Victor Bultó, President of Novartis U.S., said the approval “redefines SMA care with the first gene replacement therapy for this challenging disease, and now we can help address unmet needs across an even broader SMA population.” He added that the one‑time therapy “offers the potential to reduce the burden that comes with chronic treatment.”
John W. Day, MD, PhD, Professor of Neurology and Pediatrics at Stanford University School of Medicine, noted that the approval “is a game‑changing advance, expanding the use of transformational gene replacement therapy for SMA across age groups and signaling new possibilities for the broader field of neurological disorders and genetic medicine.”
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