FDA Approves First-Line Therapy for Neuroblastoma

FDA logo
FDA logo
The FDA fast-tracked the new drug for its safety and effectiveness in the treatment of a rare pediatric disorder.

The FDA has approved dinutuximab (Unituxin), a first-line therapy for high-risk neuroblastoma, a cancer that typically occurs in children. 

Unituxin is an antibody that binds to the surface of neuroblastoma cells, and is approved as part of a multimodality regimen that may include surgery, chemotherapy, and radiation therapy for patients that have had at least a partial response to first-line multimodality therapy. 

The therapy is the first to specifically target neuroblastoma, which occurs in approximately one in 100,000 children in the U.S. and is slightly more common in boys. Patients diagnosed with high-risk neuroblastoma have a 40 to 50% chance of long-term survival even with aggressive therapy. 

Unituxin was evaluated in a clinical trial of 226 pediatric patients with high-risk neuroblastoma whose tumors shrunk or disappeared after treatment with chemotherapy and surgery as well as additional intensive chemotherapy, bone marrow transplantation support, and radiation therapy. Participants received either oral isotretinoin (RA) or Unituxin in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor, which is believed to enhance the activity of Unituxin by stimulating the immune system and RA. 

Three years after treatment, 63% of participants that received Unituxin combination were alive and free of tumor growth or recurrence compared to 46% of participants that received RA treatment alone. An update showed that 73% who received Unituxin combination were alive compared to 58% of those who received RA alone. 

Unituxin was granted a priority review and orphan product designation by the FDA in order to shorten the review time frame since the drug treats a serious pediatric condition

The new therapy does carry a boxed warning that alerts users and health care professionals that Unituxin irritates nerve cells, which can cause severe pain and may require treatment with intravenous narcotics. The drug can also cause nerve damage and life-threatening infusion site reactions, including upper airway swelling, difficulty breathing, and low blood pressure either during or after infusion. The therapy may also cause infections, eye problems, electrolyte abnormalities, and bone marrow suppression. 

Common side effects include severe pain, fever, low platelet counts, infusion reactions, low blood pressure, hyponatremia, elevated liver enzymes, anemia, vomiting, diarrhea, low blood potassium, capillary leak syndrome, low white blood cell count, hives, and low calcium levels.