Novartis Reports Nine Tumor Cases After Zolgensma Treatment

The Swiss drugmaker said it notified health authorities in each case and would continue working with them on next steps. “Patient safety is our priority,” Novartis said.

Zolgensma is a one-time treatment for spinal muscular atrophy (SMA), a rare muscle-wasting disease affecting infants. Gene therapies such as Zolgensma deliver genetic material intended to address the underlying cause of a disease.

Novartis said the potential risk of tumors arising from integration of the delivered genetic material is already included in Zolgensma’s prescribing information. More than 6,000 patients have received the treatment through clinical trials, managed-access programs and commercial use, the company said.

SMA Europe, an organization representing SMA patient groups, said it was aware of the tumor reports involving children previously treated with Zolgensma. It said it understood that relevant regulatory authorities were actively involved.