The US Food and Drug Administration (FDA) has approved Novartis' Itvisma (onasemnogene abeparvovec-brve), making it the first and only gene replacement therapy available for the broad population of children two years and older, teens, and adults living with Spinal Muscular Atrophy (SMA) who have a confirmed mutation in the survival motor neuron 1 (SMN1) gene.

SMA is a rare, genetic neuromuscular disease where the body is unable to produce enough SMN protein due to a missing or mutated SMN1 gene, leading to the irreversible loss of motor neurons and progressive muscle weakness.

Itvisma is uniquely designed as a one-time, fixed-dose intrathecal injection to directly address this root cause by providing a functional copy of the SMN1 gene, which offers the potential to reduce the burden associated with chronic, administered treatments.

The approval was based on data from the registrational Phase III STEER study and the open-label Phase IIIb STRENGTH study, which collectively demonstrated statistically significant and sustained improvements in motor function and stabilization of motor abilities over 52 weeks, regardless of a patient's prior SMA treatment history. While showing a consistent safety profile, the most common adverse events in these studies included upper respiratory tract infection, pyrexia (fever), common cold, and vomiting. Industry experts and patient advocates hailed the approval as a "game-changing advance" that expands the use of this transformational therapy across age groups and addresses a major unmet need in the older SMA community. Novartis confirmed that Itvisma will be available in the US in December, with dedicated patient support services available to assist with treatment access.