FDA expands Yervoy approval to include adjuvant treatment of stage III melanoma

The FDA expanded the approval of ipilimumab to allow its use for adjuvant treatment of patients with stage III melanoma.

The approval is intended to help reduce the risk that melanoma will recur in these patients after surgery.

Richard Pazdur

Ipilimumab (Yervoy, Bristol-Myers Squibb) — a monoclonal antibody — blocks CTLA-4, a molecule suspected of impeding the immune system’s ability to fight cancerous cells. Ipilimumab, administered intravenously, takes the “brakes” off the body’s immune system and allows it to recognize and target cells in melanoma tumors.

The agent received FDA approval in 2011 for treatment of unresectable late-stage melanoma.

“[This] approval of Yervoy extends its use to patients who are at high risk of developing recurrence of melanoma after surgery,” Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, said in a press release. “This new use of the drug in earlier stages of the disease builds on our understanding of the immune system’s interaction with cancer.”

Stage III melanoma is characterized by disease that has reached at least one lymph node. Standard treatment is surgery to remove the melanoma skin lesions and nearby lymph nodes, according to the FDA.

The agency based the expanded approval of ipilimumab on results of a study that included 951 patients assigned to either ipilimumab or placebo as adjuvant therapy after complete surgical removal of melanoma.

Results showed a lower percentage of patients assigned ipilimumab experienced recurrence (49% vs. 62%). Researchers also reported a longer mean time to recurrence in the ipilimumab arm (26 months vs. 17 months). The OS analysis has not been completed.

The most common adverse effects reported in ipilimumab-treated patients included rash, fatigue, diarrhea, itching, weight loss, headache and nausea. The agent also may cause autoimmune disease in the digestive system, skin, liver, nervous system and hormone-producing glands, according to the FDA.

The label includes a boxed warning about the potential for fatal immune-mediated adverse reactions and unusual severe side effects.