FDA investigates tumour reports linked to Zolgensma

Novartis has reported nine cases of tumours in patients previously treated with its spinal muscular atrophy gene therapy Zolgensma, including two in which the company says testing found evidence that the therapy’s viral vector DNA had integrated into patients’ DNA. The U.S. Food and Drug Administration said it is investigating possible tumour formation associated with the treatment and assessing whether regulatory action is appropriate.

The reports concern patients treated since Zolgensma was first approved in 2019. Novartis said the nine are reports received by the company, not a complete count of tumours among all people treated. It has said Zolgensma has been used in more than 6,000 patients worldwide, including in clinical trials, managed access programs and routine care.

In the two cases with evidence of integration, doctors found that genetic material from the modified virus used to deliver a working gene copy had entered the patients’ DNA, according to Novartis. The company said both patients had received treatment and were continuing to be monitored. It has not publicly provided comprehensive details of the tumour types or the patients’ clinical courses.