EMA Committee Backs Novartis Gene Therapy Itvisma for SMA Patients Aged Two and Over

On 23 April 2026, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for Itvisma, a gene therapy for people aged 2 years and older living with spinal muscular atrophy (SMA).

This opinion will now be reviewed by the European Commission, with a final decision expected in the months.

If approved, Itvisma (intrathecal onasemnogene abeparvovec) would become the first and only gene replacement therapy in the EU for children aged two years and older living with SMA. The treatment was developed by Novartis, which also developed Zolgensma for younger children with SMA.

See the attached letter from SMA Europe for more details.