US FDA Investigates Tumor Risk Linked to Novartis Gene Therapy Zolgensma

The US Food and Drug Administration (FDA) announced on Friday that it is investigating a risk of neoplasia, which includes tumor formation and malignancy, associated with Novartis’ gene therapy Zolgensma. The regulatory scrutiny comes after the Swiss drugmaker disclosed two cases in which tumors in children showed evidence of being linked to the treatment.

Zolgensma is a one-time gene therapy used to treat children under the age of two with spinal muscular atrophy. It uses a modified virus to deliver a working copy of a gene into the patients' cells. According to Novartis, doctors found evidence in two of the cases that the carrier used in the treatment had inserted its genetic material into the patients' own DNA.

Novartis stated that it has received a total of nine reports of tumors in patients previously treated with Zolgensma since the therapy received its initial approval in 2019. The treatment has been administered to more than 6,000 children globally. The Basel-based firm maintained that it had notified health authorities in a timely and appropriate manner regarding the cases and noted that the potential risk of tumors is already included in the prescribing information for Zolgensma.

The findings have also followed complaints from patient advocates, including the organization SMA Europe, which urged Novartis to provide clearer information regarding what is known, what remains uncertain, and what the findings mean for children who have received the treatment.

Novartis manufactures two separate gene therapies for treating spinal muscular atrophy. Zolgensma is administered via intravenous infusion for pediatric patients under two years old. The second therapy, Itvisma, is administered via intrathecal injection and is used for adults and pediatric patients aged two years and older. The FDA noted that there have been no reported cases of neoplasia following the administration of Itvisma, which was approved last year for treating a rare muscle disorder.

The FDA stated that the overall benefits of both Zolgensma and Itvisma continue to outweigh their potential risks for their approved uses. However, the regulatory agency is currently evaluating whether further regulatory action is appropriate in light of the newly highlighted risks.

"This development highlights the immense regulatory and scientific complexities involved in advanced biotech and gene therapies. For businesses and investors operating in the healthcare and life sciences sector, it underscores the critical importance of transparent communication with regulatory bodies and patient advocacy groups from early development through commercialization. Long-term patient monitoring and proactive risk disclosure remain foundational to maintaining market trust and navigating regulatory oversight in high-stakes medical sectors." — Dr. Shishir Gupta, Founder & CEO, StartupLanes