On Monday, the U.S. Food and Drug Administration (FDA) approved Novartis AG’s Itvisma (onasemnogene abeparvovec-brve) for children two years and older, teens, and adults with spinal muscular atrophy (SMA).

The approval makes the gene replacement therapy the first and only one available for this broad population.

Itvisma addresses the genetic root cause of SMA. The one-time fixed dose does not need adjustment for age or body weight.

The patients with SMA have a confirmed mutation in the survival motor neuron 1 (SMN1) gene.

By replacing the SMN1 gene, Itvisma can improve motor function, offering the potential to reduce the need for chronically administered treatment associated with other available therapies for this population.

The approval of Itvisma is based on data from the registrational Phase 3 STEER study. It’s supported by the open-label Phase 3b STRENGTH study.

Also Read: Biogen-Ionis Partnered Drug Shows Benefit In Children With Spinal Muscular Atrophy Previously Treated With Novartis’ Zolgensma

Itvisma showed statistically significant improvements in motor function and stabilization of motor abilities typically not seen in the natural history of the disease, with effects sustained over 52 weeks of follow-up.

Additionally, Itvisma demonstrated a safety profile with adverse events that were consistent across both studies.

SMA is a rare, genetic neuromuscular disease caused by a mutated or missing SMN1 gene.

The SMN1 gene is responsible for producing most of the protein the body needs for breathing, swallowing, and basic movement.

Without it, motor neurons are irreversibly lost, leading to progressive muscle weakness.

Approximately 9,000 people in the US live with SMA.

In September, the FDA issued a Complete Response Letter (CRL) for Scholar Rock's ( SRRK ) apitegromab Biologics License Application (BLA) for spinal muscular atrophy (SMA).

The CRL is related to observations identified during a routine general site inspection of Catalent Indiana LLC.

Catalent is a third-party fill-finish facility which Novo Nordisk A/S acquired in December 2024.

Price Action: NVS stock is up 2.46% at $129.65 at the last check on Tuesday.

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