ZURICH, Nov 6 (Reuters) – Swiss drugmaker Novartis said on Friday that two cases of tumours in children showed evidence of being linked to its gene therapy drug Zolgensma, which is used to treat patients less than 2 years old with spinal muscular atrophy.
The company said it had received nine reports overall of tumours in patients previously treated with Zolgensma, since the therapy was first approved in 2019.
Zolgensma uses a modified virus to deliver a working copy of a gene into cells. In two of the cases, doctors found evidence that the carrier used in the treatment had inserted its genetic material into the patients’ own DNA, the company said.
The findings point to a possible link between tumours and a one-time therapy that Novartis confirmed had been given to more than 6,000 children. They also follow complaints from patient advocates that Novartis was slow to share information about the cases.
The Basel-based firm said it had notified health authorities in a timely and appropriate manner in both cases and that the potential risk of tumours is included in Zolgensma’s prescribing information.
The patient advocacy organisation SMA Europe had urged Novartis to provide information on what is known, uncertain and what it could mean for children who had received treatment.
Novartis did not provide details on where the two new cases were in its statement.
The EMA and FDA did not immediately respond to a Reuters’ request for comment.
(Reporting by Marleen Kaesebier and Bhanvi Satija;Editing by Elaine Hardcastle)
