Novartis has secured US Food and Drug Administration (FDA) approval for Itvisma (onasemnogene abeparvovec-brve), a gene replacement therapy for spinal muscular atrophy (SMA).
The clearance for this single-dose gene replacement therapy gives healthcare teams another way to manage motor-neuron mutation in older children, teens, and adults with confirmed SMA.
The FDA authorisation covers a one-time fixed-dose therapy that replaces the SMN1 gene without any need to adjust for age or body weight. Novartis says the treatment is built to address the underlying genetic cause of SMA by supplying a working copy of the SMN1 gene to help maintain motor-function stability.
The regulatory green light follows results from the open-label Phase IIIb STRENGTH study and the Phase III STEER trial, both of which reported meaningful gains in stabilisation and motor outcomes over 52 weeks. The clinical data also indicated a consistent safety profile. SMA itself stems from a missing or defective SMN1 gene, which disrupts production of SMN protein required for neuromuscular function.
Further programme details show that Itvisma is administered as a single intrathecal injection to sustain SMN protein expression, aligning with durable gene replacement therapy modalities that reduce treatment frequency. Novartis plans to introduce the product in the US market in December 2025.
“After redefining SMA care with the first gene replacement therapy for this challenging disease, we can now help address unmet needs across an even broader SMA population with the approval of Itvisma.” said Novartis US president Victor Bultó. “We are proud to support the SMA community by empowering patients of all ages through our innovative, one-time therapies, offering the potential to reduce the burden that comes with chronic treatment.”
