The drug is approved in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2), and 13-cis-retinoic acid (RA) in patients who achieve at least a partial response to prior first-line multiagent, multimodality therapy.
Neuroblastoma is the most common extracranial solid cancer in childhood and the most common cancer in infancy, with an annual incidence in the US of about 700 patients, of whom 50% are diagnosed as having high-risk disease.
Unituxin induces cell lysis of GD2-expressing cells through antibody-dependent cell-mediated cytoxicity (ADCC) and complement-dependent cytoxicity (CDC) and is part of an immunotherapeutic regimen to treat pediatric high-risk neuroblastoma.
United Therapeutics president and co-chief executive officer Roger Jeffs said: "This approval has been a collaborative effort between the National Cancer Institute (NCI), the Children’s Oncology Group and United Therapeutics for the first approved therapy for pediatric high-risk neuroblastoma.
"We are grateful for the FDA’s thorough review and collaboration on this program, and we look forward to expanding our research efforts in the area of pediatric oncology."
The FDA approval was based on demonstration of improved event-free survival (EFS) and overall survival (OS) from a multicenter, open-label, randomized trial (ANBL0032) conducted by the Children’s Oncology Group (COG).
During the trial, a total of 226 patients were randomized (1:1) to either the Unituxin/13-cis-retinoic acid (RA) arm or the RA alone arm.
The Unituxin/RA arm included Unituxin in combination with granulocyte macrophage-colony stimulating factor and RA (cycles 1, 3, and 5), Unituxin in combination with interleukin-2 and RA (cycles 2 and 4), and RA (cycle 6).