FDA approves drug for neuroblastoma

(Reuters) - The U.S. Food and Drug Administration has approved United Therapeutics Corp's drug to treat neuroblastoma, a rare cancer that typically occurs in children below 5 years.

Unituxin (dinutuximab) is the first drug approved to treat high-risk neuroblastoma patients who have a greater chance of tumors recurring or progressing after chemotherapy.

Its safety and effectiveness were tested in a study of 226 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 receive isotretinoin (RA), either alone or with Unituxin in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor, which are thought to enhance the activity of Unituxin by stimulating the immune system.

In a press statement today, the manufacturer said, "The recommended dose and schedule for Unituxin is 17.5 mg/m2/day as a diluted intravenous infusion over 10 to 20 hours for 4 consecutive days for a maximum of 5 cycles. Patients require intravenous treatment with opioids prior to, during, and for two hours following the Unituxin infusion to mitigate neuropathic pain."

The primary efficacy outcome - median event-free survival - was not reached in the Unituxin/RA arm and was 1.9 years in the RA arm.

Three years later, 63% of participants in the Unituxin arm were alive and free of tumor growth or recurrence, compared to 46% of participants treated with RA alone. In an updated analysis, 73% of those who received the Unituxin combination were alive, compared with 58% of those receiving RA alone.

A boxed warning advises that Unituxin irritates nerve cells, causing severe pain that requires intravenous narcotics and can also cause nerve damage and life-threatening infusion reactions. Unituxin may also cause other serious side effects including infections, eye problems, electrolyte abnormalities and bone marrow suppression.

There are an estimated 650 cases of neuroblastoma diagnosed in the United States each year. The disease, slightly more common in boys, forms from immature nerve cells.

The FDA press release is here: http://1.usa.gov/1GntoU3.

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