FDA approves Unituxin for Neuroblastoma-United Therapeutics Corp

The FDA has approved Unituxin (dinutuximab) Injection, from United Therapeutics Corp, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2), and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy.

Approval was based on demonstration of improved event-free survival (EFS) and overall survival (OS) in a multicenter, open-label, randomized trial conducted by the Children's Oncology Group. The trial randomized (1:1) 226 patients to either the Unituxin/13-cis-retinoic acid (RA) arm or the RA alone arm. Patients were 11 months to 15 years of age.

At the seventh interim analysis, an improvement in EFS was demonstrated and four remaining patients undergoing treatment on the RA arm crossed over to receive Unituxin/RA. The median EFS was not reached in the Unituxin/RA arm and was 1.9 years in the RA arm. An analysis of overall survival (OS) conducted three years later documented an improvement in OS in the Unituxin/RA arm compared to the RA arm. At the time of this survival analysis, median OS had not been reached in either arm. The most common serious adverse reactions (greater than or equal to 5%) are infections, infusion reactions, hypokalemia, hypotension, pain, fever, and capillary leak syndrome.

In connection with the Unituxin approval, United Therapeutics agreed to certain postmarketing requirements (PMRs) and certain postmarketing commitments (PMCs), which are studies that sponsors conduct after FDA approval to gather additional information about a product's safety, efficacy, or optimal use. PMRs are required studies, whereas a sponsor voluntarily commits to conduct PMCs.