Novartis (NYSE:NVS) said the European Medicines Agency’s Committee for Medicinal Products for Human Use has issued a favourable opinion recommending approval of Itvisma, an intrathecal gene therapy for spinal muscular atrophy (SMA).
The recommendation covers use in children aged two and above, as well as adolescents and adults with 5q SMA carrying a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene. The treatment is designed as a one-time fixed-dose therapy aimed at replacing the defective SMN1 gene.
Clinical Data Supports Efficacy
The positive opinion is based on findings from the pivotal STEER trial, alongside supporting data from the Phase IIIb STRENGTH and Phase I/II STRONG studies.
In the STEER study, Itvisma delivered a statistically significant improvement of 2.39 points on the Hammersmith Functional Motor Scale, compared with a 0.51-point gain in the control group, achieving a p-value of 0.0074. These benefits were maintained over a 52-week follow-up period.
“Itvisma met the primary endpoint showing motor function improvement versus placebo,” said Tim Hagenacker of the University Hospital Essen. “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.”
Study Details and Next Steps
The STEER trial included 75 patients treated with Itvisma and 51 patients receiving a sham procedure. Results from both STEER and STRENGTH have been published in Nature Medicine.
A final decision from the European Commission is expected within around two months following the committee’s recommendation.
Existing SMA Portfolio
Novartis already markets Zolgensma, a gene therapy approved for infants and young children with SMA, positioning Itvisma as a potential expansion of its treatment offering across a broader patient population.