The FDA has opened an investigation into Novartis’ Zolgensma (onasemnogene abeparvovec) following reports of cancer occurring in association with the spinal muscular atrophy gene therapy. The inquiry is triggered by what the agency described as a “serious risk” and comes after Novartis disclosed information earlier, prompting heightened scrutiny of long-term safety signals for at-home and pediatric gene delivery programs. For SMA gene therapy stakeholders, the development raises practical questions around pharmacovigilance expectations, case definition, and how regulators may refine risk-management plans for oncology outcomes following AAV-based treatments. Until more details emerge, clinicians and payers will likely look to follow any emerging guidance on monitoring protocols and reporting timelines.