Novartis announced that the European Commission has approved Itvisma® (onasemnogene abeparvovec) for the treatment of 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1gene. This landmark decision establishes Itvisma as the first and only one-time gene replacement therapy in the European Union (EU) for children aged two and older, adolescents, and adults.
Delivered via a single intrathecal injection, Itvisma targets the genetic root cause of the disease by introducing a functional copy of the human SMN1 gene. Unlike traditional therapies requiring lifelong, continuous dosing, Itvisma features a fixed, weight-independent dose, offering a transformative alternative for older patient populations.
The approval is backed by robust data from the pivotal Phase III STEER trial, which demonstrated a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE). Clinical benefits were sustained over 52 weeks in both treatment-naïve and pre-treated individuals. Combined with the existing infant therapy Zolgensma, Novartis now provides one-time gene therapy options across the entire SMA lifespan from newborns to adults.
Written by: Pragna Biswas
Graphics by: Pramit Hazra
