Nine reports since 2019: Novartis: Two tumor cases indicate a connection to gene therapy

The drug Zolgensma is used to treat young children with a rare muscle wasting disorder. Gene therapy works with a modified virus. According to manufacturer Novartis, there are now two tumor cases that could be related to this.

According to Novartis, two cases of tumors in children indicate a possible connection with the gene therapy drug Zolgensma. The Swiss pharmaceutical company said that since the therapy was first approved in 2019, the company has received a total of nine reports of tumors in patients who had previously been treated with Zolgensma.

In two of the cases, doctors found evidence that the carrier used in the treatment had incorporated its genetic material into the patients' own DNA, the company explained. The drug is used to treat patients under two years of age with the rare muscle wasting disease spinal muscular atrophy (SMA).

Zolgensma uses a modified virus to deliver a working copy of a gene into cells. The one-time therapy was administered to a total of more than 6,000 children. Novartis did not provide any information in its statement about where the two new cases occurred.

Complaints from patient representatives

The findings also follow complaints from patient advocates that Novartis was slow in sharing information about the cases. The patient organization SMA Europe had called on Novartis to explain what was known and what was uncertain and what this could mean for the children being treated.

The Basel-based company said it had informed the health authorities in a timely and appropriate manner in both cases. The potential risk of tumors is also included in the Zolgensma prescribing information.

The US Food and Drug Administration (FDA) now wants to investigate the risk of benign and malignant tumors in gene therapy. The authority announced on Friday that it was examining whether regulatory steps were necessary. With Zolgensma and Itvisma, another gene therapy against SMA from the Swiss pharmaceutical company approved last year, the benefits continue to outweigh the potential risks in the approved areas of application. To date, no such new tissue formations (neoplasia) have been reported for Itvisma.

The European Medicines Agency (EMA) has not yet responded to a Reuters request for comment.