FDA approves first drug for high-risk neuroblastoma



The Food and Drug Administration has approved dinutuximab as part of a multimodality regimen, including surgery, chemotherapy, and radiation therapy, for pediatric patients with high-risk neuroblastoma.

The drug “marks the first approval for a therapy aimed specifically for the treatment of patients with high-risk neuroblastoma,” Dr. Richard Pazdur, director of the office of hematology and oncology products in the FDA’s Center for Drug Evaluation and Research, said in the March 10 FDA announcement.

Dinutuximab is a chimeric monoclonal antibody that targets GD2, a glycolipid on the surface of tumor cells, according to the manufacturer, United Therapeutics.

The FDA granted approval based on a clinical trial of 226 pediatric participants 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. Three years after being randomly assigned to receive either isotretinoin or dinutuximab in combination with interleukin-2 and granulocyte macrophage colony-stimulating factor, 63% of participants receiving the combination were alive and free of tumor growth or recurrence, compared with 46% of participants treated with isotretinoin alone.

In an updated analysis, 73% of participants who received the combination were alive, compared with 58% of those receiving isotretinoin alone, the FDA said in the announcement.

The most common side effects were severe pain, fever, low platelet counts, infusion reactions, low blood pressure, hyponatremia, elevated liver enzymes, anemia, vomiting, diarrhea, low potassium levels in the blood, capillary leak syndrome, neutropenia and lymphopenia, hives, and low blood calcium levels, the statement said.

The drug carries a boxed warning that the drug can irritate nerve cells, causing severe pain that requires treatment with intravenous narcotics, and can 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. Dinutuximab may also cause other serious side effects, including infections, eye problems, electrolyte abnormalities, and bone marrow suppression.

The FDA granted approval of dinutuximab, to be marketed as Unituxin, following a priority review and orphan product designation.
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