The US Food and Drug Administration (FDA) announced today that it has expanded the indication of intravenous (IV) ipilimumab to include the treatment of pediatric patients with unresectable or metastatic melanoma who are 12 years of age and older.1
Ipilimumab is a CTLA-4 inhibitor previously indicated for patients aged 12 years and older who have undergone complete resection for cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm.
The FDA approved the indication expansion based on evidence from 2 clinical trials that evaluated the drug’s safety and efficacy in pediatric patients.
In a dose-determining trial, IV ipilimumab was administered to 33 pediatric patients with relapsed or refractory solid tumors at doses of 1, 3, 5, and 10 mg/kg over 90 minute infusions every 3 weeks for 4 cycles, then was continued every 12 weeks until progression or discontinuation.
In another trial, IV ipilimumab 3 mg/kg or 10 mg/kg was administered over 90 minutes to 12 pediatric patients with previously treated or untreated unresectable stage III or IV malignant melanoma for 3 weeks for 4 cycles.
The trials resulted in 17 patients experiencing objective responses, as well as 1 partial response that was sustained for 16 months. The approved dose is 3 mg/kg IV administered over 90 minutes every 3 weeks for 4 cycles.
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The most frequently observed adverse events associated with ipilimumab are immune-mediated and include enterocolitis, dermatitis, hepatitis, endocrinopathy, and neuropathy.