Novartis reports nine tumor cases in children treated with Zolgensma

Novartis said it has received reports of nine tumors in children previously treated with Zolgensma, its gene therapy for spinal muscular atrophy. The company said two cases showed evidence that DNA from the therapy’s viral vector had integrated into patients’ DNA. MedPage Today reported that Novartis linked those two cases to the drug. The company did not say when tumors were found in the other seven cases.

Novartis said the two patients with evidence of integration underwent treatment and continue to be monitored. BioPharma Dive reported that Cure SMA said three of the nine patients had their tumors tested, and one tested case appeared unrelated to Zolgensma. Patient groups said they are seeking information and reviewing available safety data. Novartis said the potential risk from vector integration is already included in Zolgensma’s prescribing information.