Novartis Gains EU Approval for SMA Gene Therapy

Approval Overview

European Commission approved Novartis' gene therapy Itvisma (onasemnogene abeparvovec) for treating patients with 5q spinal muscular atrophy (SMA) aged two years and older, including teenagers and adults.

Therapy Details

Itvisma is the first gene replacement therapy authorized in the EU for this broad SMA population. It is administered as a fixed one‑time dose that does not require adjustment for patient age or body weight and works by replacing the SMN1 gene to improve motor function.

Clinical Evidence

The approval is based on the registrational STEER study, which demonstrated a 2.39‑point improvement on the Hammersmith Functional Motor Scale with the benefit maintained over 52 weeks of follow‑up. Supporting data from the Phase IIIb STRENGTH study and the Phase I/II STRONG study confirmed efficacy in both treatment‑naïve and pre‑treated patients.

Statements

Nicole Gusset, CEO of SMA Europe, said the European approval is an important milestone for the SMA community and brings a new treatment option closer to patients and families. Patrick Horber, President of International at Novartis, noted that the approval enables Novartis to offer gene replacement therapy across all stages of SMA in Europe, complementing its existing Zolgensma therapy.