STAT+: Novartis cites cases of previously unknown tumors possibly tied to its SMA gene therapy

Ed’s stories explore prescription drug pricing, affordability and access, as well issues surrounding patents, litigation, and legislation. He is also the author of the morning Pharmalittle newsletter and the afternoon Pharmalot newsletter.Novartis has acknowledged seven previously unknown instances in which tumors were found in children who were treated with its spinal muscular atrophy therapy, a surprising development that has raised concerns among families, physicians, and patient advocacy groups. The cases, which for the moment are being seen as only a potential safety signal, were publicized on Thursday by an advocacy group and on a Facebook page after Novartis earlier this week disclosed the information to neurologists. One advocacy group, SMA Europe, issued a statement saying that regulatory authorities are now examining the cases. The drugmaker issued its own statement saying that since its treatment — called Zolgensma — was approved in 2019, it has received nine reports of tumors in previously treated patients. Of those, two cases to date showed evidence that the tumors may be linked to the treatment itself. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe