New Zolgensma data in SMA further demonstrate therapeutic benefit

Novartis Gene Therapies (formerly AveXis, that was acquired by Swiss pharma giant Novartis [NOVN: VX]) today announced new interim data from the ongoing Phase III STR1VE-EU clinical trial for Zolgensma (onasemnogene abeparvovec) that demonstrated patients with spinal muscular atrophy (SMA) Type 1 continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function and motor milestone achievement, including for some patients with more aggressive disease at baseline compared to previous trials.

These data as of December 31, 2019, and presented today during a virtual Clinical Trial Poster Session as part of the World Muscle Society (WMS) 2020 Virtual Congress, support the robust clinical evidence that has demonstrated a consistent, transformative benefit across the Zolgensma clinical trials for the treatment of patients with SMA.

Zolgensma is a $2.1 million-per-patient gene therapy, for which peak global sales of $2.5 billion have been forecast by GlobalData. Novartis’ shares were up 1.6% at 81.18 Swiss francs in early afternoon trading today.