The FDA has approved dinutuximab (Unituxin) for high-risk neuroblastoma in pediatric patients, the agency announced.
The monoclonal antibody was OK'd as part of first-line therapy for pediatric patients, which also includes surgery, chemotherapy, and radiation therapy for those who achieved at least a partial response to prior first-line multi-agent, multimodal therapy.
Approval follows a clinical trial of 226 pediatric patients with high-risk neuroblastoma whose tumors shrunk or disappeared after treatment with multiple-drug chemotherapy and surgery followed by additional intensive chemotherapy and who subsequently received bone marrow transplant support and radiation therapy.
Patients were randomized to either an oral retinoid drug, isotretinoin, or dinutuximab in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factors, which are thought to enhance the activity of the monoclonal antibody by stimulating the immune system.
The study showed better rates of survival and were free of disease progression over 3 years for the drug group compared with the control group (63% versus 46%).
The drug carries a boxed warning about nerve cell irritation that causes severe pain requiring treatment with intravenous narcotics. It can also cause nerve damage and life-threatening infusion reactions, including upper airway swelling, difficulty breathing, and low blood pressure during or shortly after completion of the infusion.
Other serious side effects include infections, eye problems, electrolyte abnormalities, and bone marrow suppression, the FDA said.
The most common side effects were severe pain, fever, low platelet counts, infusion reactions, low blood pressure, hyponatremia, hives, and low blood calcium levels.
The drug had been granted priority review and orphan drug designation. As an added bonus to drugmaker United Therapeutics, the firm also received a rare pediatric disease priority review voucher, allowing fast-track review on a subsequent drug application that wouldn't otherwise qualify.