Novartis acknowledges tumor cases in children treated with spinal muscular atrophy therapy

Novartis has acknowledged cases of tumors in children treated with its spinal muscular atrophy therapy. STAT and Reuters both reported the acknowledgment.

The two outlets give different figures. The reports reviewed by Vocemundi do not explain how those figures relate to each other.

According to STAT, Novartis acknowledged seven previously unknown instances in which tumors were found in children treated with the therapy. STAT reported that the company disclosed the information to neurologists earlier this week. The cases were publicized on Thursday by an advocacy group and on a Facebook page, according to STAT.

STAT also reported that, for the moment, the seven cases are being seen as only a potential safety signal. The development has raised concerns among families, physicians and patient advocacy groups, according to the outlet.

According to Reuters, Novartis said on Friday that two cases of tumors in children showed evidence of being linked to its gene therapy Zolgensma. Reuters reported that Zolgensma is used to treat patients under 2 years old who have spinal muscular atrophy.

STAT and Reuters are separate outlets, and neither credits the other. However, the information in both reports appears to trace at least partly to Novartis.

What each side says

• Novartis: Two cases of tumors in children showed evidence of being linked to Zolgensma, according to Reuters.
• SMA Europe: Regulatory authorities are now examining the cases, according to STAT.

What the reports do not show

The reports do not say whether the two cases described by Reuters are among the seven described by STAT. The available text of the STAT report does not name the therapy, so the reports do not establish that all seven cases involve Zolgensma.

The reports do not describe the evidence behind the reported link in the two cases or how that link was assessed. They do not include the tumor types, whether the tumors were malignant, or the children's ages, locations or current conditions. They also do not include how long after treatment the tumors were found or how many patients have received the therapy.

The reports do not identify which regulatory authorities are examining the cases, and they include no statement from any regulator. They do not describe any changes to the therapy's use, labeling or availability, or any further steps by Novartis. They also do not include the full text of the company's communication to neurologists or any full company statement.

Analysis

The main open question is how the seven cases and the two cases relate. A reported link in two cases and a potential safety signal do not, on their own, show how often tumors might occur or what caused them. Tumor types, timing and the number of patients treated would all help place the reports in context. If regulators are examining the cases, as SMA Europe says, any findings could clarify the picture for families and physicians. Those findings could support, narrow or weaken the concerns raised so far. The reports reviewed by Vocemundi do not indicate what any review will conclude.

Produced by the Vocemundi newsroom with AI assistance.