FDA approves drug for classic Hodgkin lymphoma patients 12 and older based on clinical trial led by researcher at UB, Roswell Park

BUFFALO, N.Y. – 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. Earlier this year, the Journal of Clinical Oncology published a more detailed subset analysis of the adolescent patients in the study, including longer follow-up data that confirmed the excellent results with nivolumab. The FDA reviewed all of these data in making its determination to approve the drug.

Kara M. Kelly, MD, division chief of hematology/oncology in the Department of Pediatrics, Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo, and chair of the Roswell Park Golisano Children’s Cancer and Blood Disorders Program, was senior author on the publication that focused on adolescents. She is also Waldemar J. Kaminski Endowed Department Chair of Pediatrics and professor of pediatric oncology at Roswell Park Comprehensive Cancer Center; Roswell Park was the regional study site through Kelly’s leadership role on the clinical trial. Sharon Castellino, MD, of the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta and Emory University School of Medicine, was first author.

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. These results, the authors concluded, support approving the drug for advanced stage Hodgkin lymphoma and lowering to 12 the minimum age for receiving nivolumab, a checkpoint inhibitor that has been described as “releasing the brakes” on the immune system.

Subsequently, the National Comprehensive Cancer Center guidelines — the international gold standard for treatment recommendations — 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 (cHL).  

“We are excited about the FDA’s decision because it facilitates access to nivolumab for patients 12 years and up,” says Kelly. “Plus, it’s the first time that the FDA approved a new drug for Hodgkin lymphoma in pediatric patients at the same time that they approved it for adults.”

She notes that with most cancer drugs, it has historically taken an average of six years after approval in adults for a drug to be approved in children.

“This FDA approval for a drug to treat a childhood cancer is just the most recent demonstration of how the research that Dr. Kelly and her colleagues conduct translates into very real improvements in the lives of children with cancer,” says Benny Joyner, MD, A. Conger Goodyear Professor and Chair of the Department of Pediatrics in the Jacobs School.

He notes that while only about 10% of new drugs that go through clinical trials are ultimately approved, in the case of pediatric cancer, that number is at best about 5%. “We could not be more proud of Dr. Kelly’s track record, which is fundamentally improving outcomes in pediatric cancers,” he says.

Another clinical trial that Kelly helped to spearhead, called CheckMate 744, has led to the European Medicines Agency (EMA), the European version of the FDA, approving nivolumab for patients ages 5-30 years with relapsed Hodgkin lymphoma. Roswell Park was one of the sites in the international trial. That study was sponsored by Bristol-Myers-Squibb and run through the Children’s Oncology Group network and the European Pediatric Hodgkin lymphoma network. Information from Bristol-Myers-Squibb on both clinical trials is available online.