Novartis confirms 2 tumor cases appear tied to SMA gene therapy
Advocates urge patients who received Zolgensma to contact healthcare team
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- Novartis confirmed at least two tumor cases appear tied to treatment with the SMA gene therapy Zolgensma.
- Advocacy groups urge patients treated with Zolgensma to contact their healthcare providers.
- Zolgensma is widely approved for infants and young children with SMA.
Novartis has confirmed that there have been at least two reports of tumors that appear to have been caused by treatment with Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy that is widely approved for infants and young children with spinal muscular atrophy (SMA).
Details about these cases remain murky, and advocacy organizations are urging SMA patients who have received Zolgensma to get in touch with their healthcare providers to discuss next steps in their particular situation.
“If you or your child has received Zolgensma and you have questions or concerns, please talk with your SMA care team or treating physician,” Cure SMA said in a statement. “They know your individual history and are best placed to guide decisions about care and monitoring at this time. Please do not stop or change any part of your care plan without first speaking with your healthcare provider.”
The Muscular Dystrophy Association (MDA) echoed this sentiment in a separate statement: “Limited information is available currently about the timing of events, nature of the tumors, and any future monitoring of treated individuals that may become appropriate. … We encourage families with immediate questions or concerns about treatment history, potential risks, or recommended monitoring to speak with their healthcare providers, who can offer guidance based on individual circumstances,” the group said.
Treatment’s prescribing information notes risk of tumors
SMA is chiefly caused by mutations in the SMN1 gene. Zolgensma is a gene therapy designed to deliver a healthy copy of this gene to the body’s cells, thus addressing the root cause of the disease. Zolgensma is approved in the U.S. for SMA patients younger than 2; it is also widely approved for young children with SMA in the rest of the world.
It has long been known there’s a theoretical risk that a gene therapy that works by integrating into the human genome could cause cancer — any random change to DNA can inadvertently trigger a cancer-causing mutation. Zolgensma, which is delivered in viral carriers, works by introducing circular DNA outside the cell’s own genetic material. However, the treatment’s prescribing information has noted the risk of tumors due to random integration of viral vector DNA into the genome.
In a statement reported by SMA Europe, Novartis confirmed that the risk is no longer just theoretical. Out of more than 6,000 patients worldwide who have received Zolgensma, Novartis said it has received nine reports of patients developing tumors. In two of these cases, testing showed evidence of vector DNA integration — which basically means there were tumor-driving changes in DNA that appear to have been triggered directly by Zolgensma. The company said these two patients have undergone treatment and are continuing to be monitored.
A third case, meanwhile, seems to be unrelated to Zolgensma treatment. In the other six cases, it remains unclear whether Zolgensma appears to have contributed to the cancer.
We know that this information may be deeply concerning for families, particularly for parents whose children have already received Zolgensma or who are currently considering treatment. Families need clear, reliable and understandable information and should not be left to piece together a potential safety concern from social media, rumours or incomplete reports.
Novartis said it has communicated information about these tumor cases to relevant healthcare authorities and is working with regulators to determine appropriate next steps.
Cure SMA, the MDA, SMA Europe, and SMA UK all said they are also working to monitor the situation and will keep the community informed as soon as more information is publicly available. The advocacy organizations urged families with concerns to get in touch with their own providers to discuss their specific situation.
“We know that this information may be deeply concerning for families, particularly for parents whose children have already received Zolgensma or who are currently considering treatment. Families need clear, reliable and understandable information and should not be left to piece together a potential safety concern from social media, rumours or incomplete reports,” SMA Europe stated. “We understand that the relevant regulatory authorities are actively involved and are currently assessing the available evidence and determining what further steps may be needed. We trust in the regulatory processes established to evaluate safety signals and to take the necessary measures to protect patients and ensure the continued safety of medicines on the market.”
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