Swiss pharmaceutical company Novartis AG (SIX:NOVN) (NYSE:NVS) announced on Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending marketing authorisation for Itvisma (intrathecal onasemnogene abeparvovec).

This recommendation covers treatment of children aged two years and older, teenagers, and adults with 5q spinal muscular atrophy (SMA) caused by a bi-allelic mutation in the SMN1 gene.

If approved, Itvisma would become the first and only gene replacement therapy available in the European Union for this patient population. The therapy is designed as a one-time fixed dose that replaces the SMN1 gene without adjustment for age or body weight, with the potential to reduce reliance on chronically administered treatments.

The CHMP opinion is supported by data from the Phase III STEER study as well as the Phase IIIb STRENGTH and Phase I/II STRONG studies. In STEER, Itvisma delivered a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale over 52 weeks, with results indicating sustained benefit.

Across STEER and STRENGTH, the treatment demonstrated clinically meaningful improvements in both treatment-naïve and pre-treated patients, with findings published in Nature Medicine.

The European Commission is expected to deliver a final decision within approximately two months.