Novartis Gene Therapy Itvisma Gets EU Approval for Spinal Muscular Atrophy

The European Commission has approved NVS's Itvisma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA), making it the first and only gene replacement therapy authorized in the EU for children two years and older, teens, and adults living with 5q SMA and a bi-allelic mutation in the SMN1 gene. The decision is backed by data from the registrational STEER study and the supportive phase 3b STRENGTH and phase 1/2 STRONG trials.

Itvisma delivers the same active ingredient as Novartis' existing therapy Zolgensma, but through a single intrathecal injection directly into the spinal canal rather than an intravenous infusion, at a fixed dose that does not need to be adjusted for age or body weight. That distinction matters commercially because Zolgensma remains restricted to infants generally under 21 kilograms due to liver-toxicity risk, leaving older children, teens, and adults with SMA without a one-time gene-replacement option until now. The approval follows closely on the heels of the FDA's approval of a CRISPR-based therapy for sickle cell disease and transfusion-dependent β thalassemia , underscoring the accelerating pace of rare-disease gene therapy approvals globally.

For Novartis, Itvisma extends the commercial life of the onasemnogene abeparvovec franchise into a previously unaddressed patient population. In the US, Itvisma carries a list price of $2.59 million, above Zolgensma's original $2.1 million price tag, and European pricing could depend on individual country reimbursement negotiations that may take months to finalize. Investors may watch uptake among older SMA patients and payer willingness to cover the therapy as key signals of how much incremental revenue the expanded label can generate.