News & Press

FDA Expands Approval of Ipilimumab For Pediatric Melanoma Patients

Last week the United States Food and Drug Administration (FDA) expanded approval of Yervoy (ipilimumab) to include pediatric patients ages 12 years and older with unresectable or metastatic melanoma. Yervoy, a monoclonal antibody that activates the immune system by blocking the CTLA-4 receptor, was first approved in 2011 to treat unresectable or metastatic melanoma in adults. In 2015, the FDA approved its use as adjuvant therapy for adult patients with Stage III cancer that had been removed surgically. This latest announcement represents a significant expansion into using immunotherapy to treat pediatric melanoma. The Melanoma Research Foundation (MRF) and the leading physicians and dedicated parent volunteers of its Pediatric Melanoma Steering Committee applaud this action as an encouraging step towards more effective treatment options and better outcomes for pediatric melanoma patients and their families.

Pediatric melanoma is a rare disease that affects approximately 500 children per year in the United States, though this number is on the rise. While there has been substantial progress in understanding the biology and genetic subtypes of adult melanoma leading to approval of targeted therapies and immunotherapies, the comparatively small number of pediatric patients has made it difficult to conduct clinical trials that can lead to new treatment options. As the incidence of melanoma in children continues to increase, the need for new efforts and strategies in prevention and research grows more urgent. 

Brenda Busby, mother to a 12-year-old patient and the MRF’s Pediatric Program Coordinator, explains the importance and context of this advance: “The ground has been broken and a solid foundation laid. We now have to raise up the building. Then another, and another, until we build the one that houses a cure.”

"The approval of ipilimumab for pediatric melanoma patients is a significant milestone because it shows children under 18 represent a growing number of melanoma cases,” says Dr. Vernon Sondak, Department Chair, Cutaneous Oncology at H. Lee Moffitt Cancer Center and member of the MRF Scientific Steering Committee. “We hope this is only the starting point, so that in the future all melanoma patients – regardless of age – have access to all the latest treatments."

“When you are told that your child has cancer and that treatment options are limited, it feels like being thrust into the fight of your lives with no weapons to fight back,” says Busby. “As someone who has lived with the many challenges of pediatric cancer, I know how important it is for patients and their families who face metastatic melanoma to have access to new therapies.”

The challenges of pediatric melanoma may have delayed progress but each new breakthrough is not only a step forward but an opportunity to accelerate the pace. With the help of the melanoma community, the MRF is committed to continue to develop new resources for patients and their families; fund cutting edge research; work with industry partners, the FDA and investigators to identify new collaborations to generate data on drugs in rare populations and to strongly advocate for important policies like increased federal research funding, an under-18 tanning ban and access to sunscreen in schools. Treatment advances are an encouraging step in the right direction, but more work remains to be done. The MRF will continue to lead the fight against melanoma on all fronts, for all patients, until the day there is a cure.

The MRF’s pediatric melanoma program is both for, and made possible by, our incredible community. We invite you to support this work on behalf of our youngest melanoma warriors.