The Food and Drug Administration (FDA) has approved Unituxin (dinutuximab; United Therapeutics), the first therapy to be approved for pediatric patients with high-risk neuroblastoma for use as part of a multimodal regimen including surgery, chemotherapy and radiation therapy for patients who achieved at least a partial response to prior first-line multiagent, multimodality therapy.
The FDA approval is based on 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 transplantation support and radiation therapy. Patients were randomly assigned to Unituxin or an oral isotretinoin (RA) in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor. Sixty-three percent of participants receiving Unituxin were alive and free of tumor growth or recurrence three years after treatment assignments vs. 46% of patients treated with RA alone. An updated analysis of survival showed 73% of the group receiving Unituxin combination survived vs. 58% of the RA alone arm.
Unituxin carries a Boxed Warning alerting patients and health care professionals that Unituxin irritates nerve cells, causing severe pain that requires treatment with intravenous narcotics and can also cause nerve damage and life-threatening infusion reactions, including upper airway swelling, difficulty breathing, and low blood pressure, during or shortly following completion of the infusion. It may also lead to other serious side effects including infections, eye problems, electrolyte abnormalities, and bone marrow suppression.
Unituxin was previously granted Priority Review and Orphan Drug designation by the FDA.
For more information visit FDA.gov.