FDA Approves Nivolumab for Pediatric Hodgkin Lymphoma, Clears Two Investigational Drugs for Trials

The Food and Drug Administration has approved the immune checkpoint inhibitor nivolumab in combination with chemotherapy for adults and adolescents 12 and older with previously untreated Stage III/IV classic Hodgkin lymphoma. The approval was based on data from a randomized clinical trial published in the New England Journal of Medicine in October 2024, with a more detailed subset analysis of adolescent patients published in the Journal of Clinical Oncology earlier this year. This marks the first time the FDA has approved a new drug for Hodgkin lymphoma in pediatric patients at the same time it was approved for adults, historically taking an average of six years after adult approval for pediatric approval.

The trial found that teens and adults with advanced-stage Hodgkin lymphoma survive longer without their disease coming back when they receive the immunotherapy nivolumab (brand name Opdivo) instead of the immunotherapy brentuximab vedotin (brand name Adcetris) along with chemotherapy. Subsequently, the National Comprehensive Cancer Center guidelines for both adult and pediatric Hodgkin lymphoma were updated to reflect the newly discovered best practice of combining nivolumab with a chemotherapy combination called AVD for adolescents and adults newly diagnosed with stage 3-4 classic Hodgkin lymphoma.

In separate regulatory actions, the FDA has cleared investigational new drug applications for two different therapies. The agency cleared an IND for FG001, allowing a U.S. phase 2 registration-oriented trial in patients with high-grade glioma, a serious form of brain cancer. Enrollment of the first patient in the U.S. trial is expected in the second quarter of 2026. FG001 is designed to light up cancer tissue during surgery to improve precision, with the goal of reducing local recurrence after surgery and lessening surgical sequelae.

The FDA also approved an Investigational New Drug application for FRF-001, a viral gene therapy designed to address the underlying genetic cause of FOXG1 syndrome, a rare neurodevelopmental disorder marked by cognitive and physical disabilities and epilepsy. FRF-001 delivers a functional copy of the FOXG1 gene using an adeno-associated virus 9 vector, making it the first FOXG1 AAV9 gene replacement therapy. The upcoming first-in-human clinical trial will be conducted across multiple sites and is being independently sponsored by the FOXG1 Research Foundation, which has secured $14.5 million so far to advance FRF-001 through clinical trials.

For the FG001 program, the company reported that it received FDA alignment on the design of its U.S. phase 2 clinical trial in high-grade glioma to support registration, as well as alignment on key elements of a subsequent phase 3 program. This regulatory agreement on trial design is intended to support a clearer path toward a future new drug application submission. The company anticipates that two U.S. clinical trials will be required to support U.S. regulatory approval of FG001 for high-grade glioma, with both trials expected to enroll patients over approximately one year.

FOXG1 syndrome is caused by mutations in the Forkhead Box G1, one of the most important genes for early brain development, affecting one in 30,000 individuals worldwide. Researchers have previously demonstrated that their drug can reverse some brain abnormalities in mice with FOXG1 syndrome, including in parts of the brain associated with language, memory and social interaction. The foundation believes this is the first instance of a parent-led rare disease nonprofit foundation independently sponsoring its own multi-site, international gene therapy clinical trial.