This March, the U.S. Food and Drug Administration (FDA) approved Unituxin (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma. There have been few treatment options available for this pediatric cancer and researchers have been working on a new drug that can help kids’ immune system to fight cancer. There were very few drugs approved that were specifically developed to treat children with cancer. Unituxin marks the first approval for a therapy that aimed specifically for treating kids with high-risk neuroblastoma.
Neuroblastoma is a rare type of cancer typically found in children younger than five years of age. As per the National Cancer Institute, neuroblastoma occurs in one out of 100,000 children and it is slightly more common in boys. Also, around 650 new cases of neuroblastoma are diagnosed in the United States every year. According to the FDA press release, neuroblastoma forms from immature nerve cells usually beginning in the adrenal glands and may also develop in the abdomen, chest or in the nerve tissue near the spine. Unituxin is an antibody that attaches to the surface of neuroblastoma cells. It is being approved for use as part of a multimodality regimen, such as surgery, chemotherapy and radiation therapy for patients who achieved at least a partial response to prior first-line multiagent, multimodality therapy.
There is only 40 to 50 percent chance of long term survival for patients with high-risk neuroblastoma despite aggressive therapy. However, Unituxin provides a treatment option that prolongs survival in pediatric patients with high-risk neuroblastoma. During the FDA clinical trials, 226 pediatric patients who had high-risk neuroblastoma and responded well to chemotherapy followed by surgery and radiotherapy were randomly assigned to receive an oral retinoid drug, isotretinoin (RA), or Unituxin in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor. After three years of treatment assignment, 63 percent of participants receiving Unituxin combination were found to be alive and free of tumor growth or recurrence, compared to 46 percent of participants treated with RA alone.
An updated analysis of survival found 73 percent of participants who received the Unituxin combination alive compared to 58 percent of those receiving RA alone.
However, Unituxin carries a boxed warning that alerts patients and healthcare professionals regarding its risks. Unituxin irritates nerve cells and may cause severe pain that requires treatment with intravenous narcotics. It may also cause nerve damage and serious infusion reactions. Other serious side effects include infections, eye problems, electrolyte abnormalities and bone marrow suppression. The most common side effects are fever, low platelet counts, infusion reactions, low blood pressure, low levels of salt in the blood, elevated liver enzymes, anemia, vomiting, diarrhea, low potassium levels in the blood, capillary leak syndrome, low numbers of infection-fighting white blood cells, hives, and low blood calcium levels.
Physicians are required to thoroughly assess patient history and current condition and make the correct diagnosis to order this drug as well as take proper care of patients after giving the drug. On medical claims, neuroblastoma (given below) is reported using the following diagnosis codes.
- 194.0: Malignant neoplasm of adrenal gland
- C74.90: Malignant neoplasm of unspecified part of unspecified adrenal gland
Drug administration is very important. It is recommended that Unituxin is given as an intravenous (IV) infusion, over 10 to 20 hours for 4 consecutive days for up to 5 cycles.