First therapy for pediatric high-risk neuroblastoma approved by FDA

March 10, 2015

FDA approved dinutuximab (Unituxin, United Therapeutics) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a type of cancer that most often occurs in young children.

FDA approved dinutuximab (Unituxin, United Therapeutics) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a type of cancer that most often occurs in young children.

According to the American Cancer Society, neuroblastoma is a type of cancer that starts in certain very early forms of nerve cells found in an embryo or fetus. (Neuro refers to nerves, while blastoma refers to a cancer that affects immature or developing cells). This type of cancer occurs most often in infants and young children. It is rarely found in children older than 10 years.

Neuroblastoma is by far the most common cancer in infants (less than 1 year old). It accounts for about 6% of all cancers in children. There are about 700 new cases of neuroblastoma each year in the United States. This number has remained about the same for many years. The average age at the time of diagnosis is about 1 to 2 years. In rare cases, neuroblastoma is detected by ultrasound even before birth. Nearly 90% of cases are diagnosed by age 5. Neuroblastoma is very rare in people over the aged 10 years. In about 2 of 3 cases, the disease has already spread to the lymph nodes or to other parts of the body when it is diagnosed.

Unituxin is an antibody that binds to the surface of neuroblastoma cells. Unituxin is being approved for use as part of a multimodality regimen, including surgery, chemotherapy and radiation therapy for patients who achieved at least a partial response to prior first-line multiagent, multimodality therapy.

“The approval of medications such as Unituxin to treat rare cancers demonstrates the ongoing effort to not only increase awareness of the condition but ensure that patients with these conditions are also receiving life-saving medications or medications that can improve their quality of life,” said FormularyWatch Advisor Abimbola Farinde, PharmD, MS, who serves on the faculty at Columbia Southern University, Orange Beach, Ala.

FDA granted Unituxin priority review and orphan product designation.

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FDA also issued a rare pediatric disease priority review voucher to United Therapeutics, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. This is the second rare pediatric disease priority review voucher granted by FDA since inception of the rare pediatric disease review voucher program, which is designed to encourage development of new therapies for prevention and treatment of certain rare pediatric diseases.

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According to an FDA press release, the safety and efficacy of Unituxin were evaluated in 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. Participants were randomly assigned to receive either an oral retinoid drug, isotretinoin (RA), or 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, and RA.

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

Unituxin carries a Boxed Warning alerting patients and healthcare professionals that Unituxin irritates nerve cells, causing severe pain that requires treatment with intravenous narcotics and can also 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. Other other serious side effects Unituxin can cause include infections, eye problems, electrolyte abnormalities and bone marrow suppression.

Severe pain, fever, low platelet counts, infusion reactions, low blood pressure, low levels of salt in the blood (hyponatremia), elevated liver enzymes, anemia, vomiting, diarrhea, low potassium levels in the blood, capillary leak syndrome (which is characterized by a massive leakage of plasma and other blood components from blood vessels into neighboring body cavities and muscles), low numbers of infection-fighting white blood cells (neutropenia and lymphopenia), hives, and low blood calcium levels, were the most common side effects.

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