Zolgensma safety investigation
Zolgensma tumor and cancer reports: what is known, and what families can do.
On November 6, 2026, the FDA announced a safety investigation after tumors were reported in children treated with Zolgensma (onasemnogene abeparvovec), the Novartis gene therapy for spinal muscular atrophy (SMA). This page puts the confirmed facts for families in one place. We update it whenever regulators or Novartis release something new.
Published November 10, 2026. Last updated November 10, 2026. Written by Tim Kingsbury, trial attorney.
Free and confidential. We do not give medical advice, and we are not telling any family to stop or avoid treatment. Those decisions belong with you and your child's care team.
The facts
Does Zolgensma cause tumors or cancer?
In a small number of children, yes, according to the FDA. In November 2026 the agency reported that tumor tissue from two children treated with Zolgensma contained the therapy's DNA, inserted at a cancer-driving gene called GLI1. The FDA calls these confirmed cases of product-related insertional oncogenesis. Nine tumor reports have been made among more than 6,000 treated children, and how large the risk is remains under investigation. [1] [2]
Much of what families have heard came through social media and doctor's office conversations. Here is what the FDA, Novartis, and patient organizations have said on the record, and what they have not.
Where the nine tumor reports stand
Each circle is one child. Testing is complete for three of the nine. [3]
2 linked to the therapy by testing
1 appears unrelated
6 testing not complete
Confirmed
- Novartis has received nine reports of tumors in children previously treated with Zolgensma. [2]
- Testing is complete on three of the nine tumors. Two were found to be related to Zolgensma. One appears unrelated. [3]
- In the two related cases, lab analysis found the therapy's DNA inside the tumor, inserted at a cancer-driving gene called GLI1. [1]
- The reported tumors involved the brain and nerves, the spine and nearby tissue, and the kidney. [1]
- The FDA says the same risk applies to Itvisma, the newer spinal injection form of the therapy. No tumors have been reported after Itvisma. [1]
- The FDA says the overall benefits of both treatments continue to outweigh their potential risks. [1]
Not known yet
- Where the nine children live, how old they are, and how they are doing. Novartis says the two children with confirmed cases were treated and are being monitored. [2]
- What kinds of tumors the other six children have, and whether the therapy played any role.
- How long after treatment a tumor can appear.
- Whether doctors will recommend routine scans for treated children.
- When Novartis first learned of each case.
How it happens
How can a gene therapy lead to a tumor?
Zolgensma works by delivering a gene. The concern is where a piece of that delivered DNA can end up.
1. The gene is delivered.
The therapy uses an engineered virus, called AAV9, to carry a working copy of the SMN1 gene into the child's cells. [5]
2. It is meant to stay separate.
The delivered DNA is designed to sit apart from the child's own DNA. In most cells, that is what happens. [5]
3. Sometimes it inserts.
A piece of the delivered DNA can insert into the child's own DNA. In the two confirmed cases, it landed at a gene called GLI1 and switched it on. An overactive GLI1 gene can drive tumor growth. [1]
Simplified illustration for general understanding. It is not medical advice.
The warning
What did the Zolgensma label say about tumor risk?
Every prescription drug has an official label that doctors rely on. The Zolgensma label has a section on this risk. It calls the risk theoretical. It acknowledges that tumors have been reported since approval. Then it says this:
That section does not recommend scans or any other screening. The FDA is now reviewing whether that language, and the long-term follow-up studies behind it, are adequate. [1]
That is the question we are examining: what the manufacturer knew about these cases, when it knew, and what it told the doctors who relied on the label.
For parents
What should parents do if their child received Zolgensma?
STEP 1
Start with your child's SMA care team.
Cure SMA advises families with questions to talk with their care team or treating physician. Bring this page if it helps. Do not change or stop any treatment on your own. [3]
STEP 2
Ask what monitoring makes sense for your child.
The FDA has asked providers to watch treated children for tumors but has not set a screening schedule. The national team that oversees Zolgensma in England currently recommends no extra scans and says it will share more as it learns more. Tell your child's doctors about any new or changing symptoms. [1] [4]
STEP 3
If your child has a tumor, ask the hospital to keep the tissue.
Testing tumor tissue is how the two confirmed cases were identified. Retention practices vary by hospital. A written request asks the pathology department to preserve what it has. You can also ask about testing that is independent of the manufacturer.
Get the tissue preservation letterSTEP 4
Keep your records together.
Note the date and hospital where your child received Zolgensma. Keep pathology and imaging reports and the names of treating doctors. Dates matter in these cases.
Timeline
Timeline of the Zolgensma tumor reports and FDA investigation
Newest first. We add an entry each time something is confirmed.
Nov. 6, 2026
The FDA announces it is investigating a serious risk of tumors linked to Zolgensma and is weighing regulatory action. [1]
Nov. 5, 2026
Novartis confirms nine tumor reports, two with evidence that the therapy's DNA entered the child's own DNA. Families had begun hearing the news from their neurologists days earlier. [2]
Nov. 4, 2026
The national team that oversees Zolgensma in England publishes a safety update based on information from Novartis. [4]
Oct. 14, 2026
The New England Journal of Medicine reports a tumor in a child treated with a different gene therapy that uses a similar delivery virus. The virus's DNA was found in the same gene, GLI1. [6]
Nov. 24, 2025
The FDA approves Itvisma, a spinal injection form of the same therapy, for patients 2 and older. [1]
2023 and 2025
Two case reports describe tumors in children treated with Zolgensma. Neither concludes the therapy was the cause. In the first, the analysis was limited by how little tissue remained. [7] [8]
May 24, 2019
The FDA approves Zolgensma for children under 2 with spinal muscular atrophy. [1]
Legal options
Is there a Zolgensma lawsuit, and can families file a claim?
As of November 9, 2026, we have not found any injury lawsuit filed in the United States over these tumor reports. There is no class action and no settlement. Humphrey, Farrington & McClain is investigating claims against the manufacturer now.
Whether your family has a claim depends on the facts. We will tell you where you stand.
If your child was diagnosed with a tumor after Zolgensma, we want to hear from you. Three things matter most at the start: the diagnosis, the date of treatment, and whether tumor tissue is available for testing.
If your child was treated and has no tumor, there is likely no claim to bring today. You can still ask us questions, and you can sign up below for updates as the investigation develops.
Our review focuses on the manufacturer. Every case is different, and no lawyer can promise a result.
Every state sets deadlines for bringing claims. Some are short, and the rules for children often differ from the rules for parents. It costs nothing to ask.
Why HFM
Why would a family bring this to us?
Humphrey, Farrington & McClain, P.C. (HFM) is a plaintiffs' trial law firm in Independence, Missouri, in the Kansas City area. We represent families across the United States in product liability, pharmaceutical, and toxic exposure cases against large manufacturers.
Novartis is one of the largest drug companies in the world. A case against it calls for a firm that has broken new ground, has handled a childhood cancer case, and has taken on companies this size. Here is our record on each.
We have gone first before.
In 1997, Ken McClain of our firm settled what was reported as the first individual tobacco injury case in the country, and won an order requiring a tobacco company to turn over documents it had kept privileged. A year later, Missouri chose the firm as co-counsel in its case against the tobacco industry. [10]
In 2001 we began representing workers whose lungs were damaged by butter flavoring. [11]
We have represented a child with cancer.
In 2025, our team reached a $30 million settlement for two salon workers exposed to formaldehyde in a professional hair-smoothing product. One was pregnant during the exposure, and her child was later diagnosed with leukemia. Missouri Lawyers Media named it one of the state's top settlements of the year. Tim Kingsbury was one of the four attorneys on the case. [9]
We know how hard a childhood cancer case is on a family, and what it takes to prove where the cancer came from.
We take on companies this size.
We have experience in pharmaceutical cases, and we have tried cases against tobacco companies and chemical and flavoring manufacturers. Results include a $90 million verdict in a PCB contamination case and more than $104 million in jury verdicts for workers injured by flavoring chemicals. [11] [12]
We prepare every case as if it will be tried.
We are selective, and that is the point. We accept a small number of serious cases each year.
Past results afford no guarantee of future results. Every case is different and must be judged on its own merits.
Common questions
Questions parents are asking, answered plainly.
Does this mean my child will develop a tumor?
No one can say that. Nine reports have been made among more than 6,000 treated children, and testing has tied two of them to the therapy so far. The FDA is still working out how large the risk is. [1] [2]
Should my child have an MRI or other scans?
That is a medical decision for your child's doctors. The FDA has told providers to monitor treated children for tumors, but it has not issued a screening schedule. Ask your neurologist what they recommend for your child and why.
Should we avoid Zolgensma or Itvisma?
We cannot answer that, and no law firm should. SMA is a serious disease, and the FDA says the benefits of both treatments still outweigh the risks. Other approved treatments exist. Your child's neurologist can walk you through the options. [1]
What do GLI1 and insertional oncogenesis mean?
Zolgensma uses an engineered virus, called AAV9, to carry a working gene into a child's cells. The delivered DNA is meant to stay mostly separate from the child's own DNA. Insertional oncogenesis is what happens when delivered DNA instead inserts into the child's DNA at a spot that switches on a cancer-driving gene. GLI1 is the gene involved in the confirmed Zolgensma cases. [1] [5]
Who makes Zolgensma?
Novartis Gene Therapies, Inc., formerly known as AveXis, a unit of the Swiss drug company Novartis. The same company makes Itvisma. [5] [13]
Is there a Zolgensma class action lawsuit?
No. As of November 9, 2026, we have not found a class action, a settlement, or any injury lawsuit over the tumor reports. Claims like these are usually brought as individual cases for each child and family.
Which law firm is handling Zolgensma tumor cases?
Humphrey, Farrington & McClain, P.C., a trial firm in Independence, Missouri, with experience in pharmaceutical cases, is reviewing claims for families anywhere in the United States whose child was diagnosed with a tumor after Zolgensma. Trial attorney Tim Kingsbury leads the review. Call (816) 836-5050 or use the form below.
What does it cost to talk with you?
Nothing. The first conversation is free and confidential. You will never owe us any money. If we take your case, we are paid only out of a recovery we win for your family. If there is no recovery, there is no fee and no bill.
We do not live in Missouri. Can you still help?
Yes. We handle cases across the country and work with a lawyer in the family's home state where needed.
Free and confidential
Tell us what happened.
If your child has been diagnosed with a tumor, a lawyer will call you back the same business day. If you only want updates or the tissue preservation letter, say so and that is all we will send.
You will never owe us any money. If we take your case, we are paid only out of a recovery we win for your family.
Prefer to call? (816) 836-5050
For referring attorneys
Co-counsel and referral inquiries are welcome. Choose "An attorney referring a family" in the form, or call and ask for Tim Kingsbury.
Sources
The facts about Zolgensma and about our firm's record come from these sources. If we get something wrong, tell us and we will fix it.
- U.S. Food and Drug Administration, FDA Investigating Serious Risk of Malignancy Linked to Zolgensma Gene Therapy, November 6, 2026.
- Novartis statement of November 5, 2026, as reported by BioSpace.
- Cure SMA, An Update for Our Community on Recent Zolgensma Safety Reports, November 5, 2026.
- SMA UK, Safety update from the National Multidisciplinary Team for Zolgensma, November 4, 2026.
- Zolgensma prescribing information, section 5.6, via DailyMed.
- Vo et al., Integration-Associated Paraspinal Neoplasm after AAV Gene Therapy, New England Journal of Medicine, October 14, 2026.
- Retson et al., Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec, Molecular Therapy, 2023.
- Holzwarth et al., Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec, Molecular Therapy, 2025.
- Missouri Lawyers Media, Hairstylists settle for $30 million in product liability case, July 15, 2025.
- Super Lawyers, If at First You Don't Succeed, Try, Try Some Cases, October 24, 2005.
- Humphrey, Farrington & McClain, Food Flavoring-Related Lung Injury.
- Humphrey, Farrington & McClain, Kenneth B. McClain, top verdicts.
- North Carolina Biotechnology Center, AveXis Renamed Novartis Gene Therapies.
Published by
Trial Attorney, Humphrey, Farrington & McClain, P.C.
Tim Kingsbury is a shareholder and trial attorney at Humphrey, Farrington & McClain, admitted to practice in Missouri and Kansas. He represents families in product liability, toxic exposure, and medical malpractice cases. He writes this page and updates it as the investigation develops.
Published November 10, 2026. Last updated November 10, 2026.