On 23 April 2026 the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for Itvisma, a Novartis intrathecal AAV9-delivered SMN1 gene therapy. The positive opinion is now under review by the European Commission, with a final decision expected in upcoming months.

Who this could help

Itvisma is intended for children aged 2 years and older, teens, and adults living with SMA — extending gene therapy access well beyond the existing infant-population label of intravenous onasemnogene abeparvovec (Zolgensma). The intrathecal delivery route reflects pivotal STEER and STRENGTH trial data designed for older patients.

Significance

If approved by the EC, Itvisma would be the first SMN-restoring gene therapy available to adolescents and adults in Europe — a population previously without single-administration genetic options. Combined with disease-modifying drugs (nusinersen, risdiplam) and the upcoming muscle-targeted approaches, it expands the treatment landscape for the full SMA age spectrum.

Source: SMA Europe May 2026 newsletter; EMA CHMP summary of opinion 23 April 2026.