FDA Approves Unituxin (Dinutuximab) for Neuroblastoma

The US Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) for children with high-risk neuroblastoma. It is the first drug approved specifically to treat this type of cancer. Neuroblastoma starts in the nervous system and most often occurs in young children.

Unituxin is a monoclonal antibody, which is a type of immunotherapy drug. It works by helping the body’s own immune system find and destroy cancer cells. It’s to be used along with other treatments, including chemotherapy, surgery, and radiation for children who have responded to previous treatment.

The approval was based on a clinical trial of 226 children with high-risk neuroblastoma who had responded to previous treatment, including a stem cell transplant. After 3 years of follow-up, 63% of children who received treatment that included Unituxin were alive and free of tumor growth or recurrence, compared with 46% of children who received treatment that did not include Unituxin. Later, an updated analysis found that 73% of children who received Unituxin were still alive, compared with 58% who did not receive Unituxin.

The FDA granted Unituxin priority review and orphan product designation, designed to speed up the approval process and to encourage development of new therapies for rare diseases.

Unituxin is given as an infusion into a vein. It carries the FDA’s strongest warning for the possibility of serious side effects. These can include irritation of nerve cells (causing severe pain that requires treatment with intravenous narcotics), nerve damage, and life-threatening infusion reactions, including upper airway swelling, difficulty breathing, and low blood pressure, during or shortly after the infusion. Unituxin may also cause other serious side effects including infections, eye problems, blood mineral abnormalities, and low blood cell counts.

The most common side effects of Unituxin are severe pain, fever, low platelet counts, infusion reactions, low blood pressure, low levels of sodium in the blood, elevated liver enzymes, anemia, vomiting, diarrhea, low potassium levels in the blood, leaking from blood vessels, low numbers of infection-fighting white blood cells, hives, and low blood calcium levels.

Unituxin is marketed by United Therapeutics.

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