Novartis (NYSE: NVS) announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion recommending marketing authorization for Itvisma, an intrathecal gene therapy for spinal muscular atrophy.

The opinion supports the treatment's use for children two years and older, teens, and adults with 5q spinal muscular atrophy containing a bi-allelic mutation in the survival motor neuron 1 gene. The therapy is designed as a one-time fixed dose that replaces the SMN1 gene.

The recommendation is based on data from the registrational STEER study and supportive Phase IIIb STRENGTH and Phase I/II STRONG studies. In STEER, Itvisma demonstrated a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale compared to 0.51 points for the sham group, with a p-value of 0.0074. The effects were sustained over 52 weeks of follow-up.

"Itvisma met the primary endpoint showing motor function improvement versus placebo," said Professor Tim Hagenacker from the Department of Neurology at University Hospital Essen in Germany. "Even a 1-point difference in the HFSME can translate into tangible functional gains for individuals with SMA, such as the ability to grasp a pen."

The STEER study included 75 patients who received Itvisma and 51 patients who received sham treatment. Results from the STEER and STRENGTH studies were published in Nature Medicine.

The European Commission is expected to issue a final decision within approximately two months following the committee's recommendation. Novartis already markets Zolgensma, another gene therapy for babies and young children with SMA.