Novartis gains FDA approval for Itvisma gene therapy to treat spinal muscular atrophy

DENMARK— Novartis has received the US Food and Drug Administration’s (FDA) approval for Itvisma (onasemnogene abeparvovec-brve), a gene replacement therapy aimed at treating spinal muscular atrophy (SMA).

This innovative therapy targets the root genetic cause of SMA and is intended for children aged two years and older, as well as teens and adults diagnosed with SMA who have a confirmed mutation in the survival motor neuron 1 (SMN1) gene.

Itvisma works by delivering a functional copy of the SMN1 gene through a single, fixed-dose intrathecal injection.

This one-time treatment improves motor function and does not require dosage adjustments based on age or body weight, simplifying the treatment process for patients and healthcare providers.

The FDA’s approval follows strong evidence from clinical trials, including the open-label Phase IIIb STRENGTH study and the Phase III STEER trial.

These studies demonstrated significant improvements in patients’ motor function and disease stabilization over a 52-week period.

Additionally, both trials confirmed that Itvisma maintains a consistent safety profile, making it a reliable option for a wide range of SMA patients.

Spinal muscular atrophy is a rare genetic neuromuscular condition caused by a defective or missing SMN1 gene.

This gene is crucial because it produces most of the survival motor neuron (SMN) protein, which is essential for proper muscle function.

Without sufficient SMN protein, patients experience muscle weakness and atrophy that progressively worsen over time.

Victor Bultó, the president of Novartis US, emphasized the company’s commitment to advancing SMA care.

He stated that, after pioneering the first gene-replacement therapy for SMA, Novartis is now expanding access to innovative treatments like Itvisma to meet the needs of a broader patient population.

Bultó highlighted the company’s goal to empower patients across all ages by offering one-time therapies that may reduce the long-term burden of chronic treatment.

Itvisma is expected to become available in the United States by December 2025, providing new hope for those living with this challenging disease.

In a related development, Novartis announced in October 2025 the acquisition of all outstanding shares of US-based Avidity Biosciences, completing the deal for about USD 12 billion in cash on a fully diluted basis.

This strategic acquisition signals Novartis’ continued investment in cutting-edge genetic therapies and innovative healthcare solutions.