The European Commission has just authorised the marketing of Itvisma (onasemnogene abeparvovec) for the treatment of SMN1-related proximal spinal muscular atrophy (SMA) in patients aged two years and over.
- This is the first gene therapy authorised in the European Union for patients with SMA aged over two years, including adolescents and adults, administered as a single dose.
- Unlike Zolgensma, which is administered intravenously and is intended for children weighing less than 21 kg, Itvisma (the same product) is administered intrathecally to older patients.
- This authorisation is based in particular on the results of the STEER and STRENGTH studies, which demonstrated a clinical benefit in terms of motor function in patients aged between 2 and 18 years.
- In France, the health authorities will examine the conditions for access to this new treatment.
