The Food and Drug Administration is investigating reports of tumors, including cancers, in patients who received Zolgensma for spinal muscular atrophy. The agency said it found evidence in two cases that genetic material from the therapy had inserted into patients’ DNA in a way that could drive tumor growth.
In its Nov. 6 safety notice, the FDA said the benefits of Zolgensma and a related treatment, Itvisma, still outweigh their potential risks for approved uses. The agency is evaluating whether to take regulatory action and says patients who receive either treatment should be monitored for tumor formation.
What the FDA found
Three reported tumors involved GLI1, a gene that can promote cancer when it becomes abnormally active. In two of those cases, tests of tumor tissue found that DNA delivered by Zolgensma had inserted within or near that gene. The FDA is still investigating what activated GLI1 in the third case. The notice did not estimate how often tumors occur after treatment.
Which treatments are involved
Zolgensma is given by intravenous infusion and is approved for children under 2 with spinal muscular atrophy. Itvisma is injected into the fluid around the spinal cord and is approved for adults and children 2 and older with the condition. Both treatments use the same carrier and genetic material. The FDA said it had received no reports of tumors after Itvisma as of Nov. 6, and it considers the potential risk relevant to both products.
The prescribing information already warns about the possibility of tumors from the insertion of the therapy’s DNA. The FDA is reviewing whether those warnings and the manufacturer’s long-term follow-up studies are adequate in light of the new findings. Health care providers can report suspected adverse reactions through FDA MedWatch or by calling 1-800-FDA-1088.
