FDA Approves Antibody for Childhood Nerve Cell Cancer

Nerve cells in brain illustration (NIH.gov)

10 March 2015. The U.S. Food and Drug Administration approved today dinutuximab, an engineered antibody to treat neuroblastoma, a rare cancer affecting nerve cells in young children. Dinutuximab is developed by United Therapeutics, a biotechnology company in Silver Spring, Maryland and marketed under the brand name Unituxin.

Neuroblastoma is a cancer that develops from immature nerve cells, and begins most often in and around adrenal glands, but can also start in other parts of the body where nerve cells exist. The cancer mainly affects children under 5 years of age, and is believed to begin before birth. About 700 cases are diagnosed in the U.S. each year, according to American Cancer Society, and account for about 6 percent of all cancer cases in children. Patients with high-risk neuroblastoma have a 40 to 50 percent chance of long term survival despite aggressive therapy.

FDA approved dinutuximab as part of a combination of treatments for high-risk neuroblastoma, including surgery, radiation, and other chemotherapies, for patients receiving at least a partial response to other treatments. Dinutuximab is an antibody designed to stimulate the immune system for breaking down cells that express molecules known as GD2 in nerve cells. GD2 is associated with a number of cancer types including neuroblastoma.

FDA based its approval in part on a late-stage clinical trial of dinutuximab in combination with other chemotherapy drugs, and after patients received chemotherapy, radiation therapy, and bone marrow transplants. The 226 patients with neuroblastoma were randomly assigned to receive dinutuximab with the other drugs believed to enhance its effects — including isotretinoin, a vitamin A compound —  or isotretinoin alone. While initial results of the trial were published in 2010, FDA was more interested in long-term survival of patients in the trial, which show 63 percent of participants taking the dinutuximab combination alive after 3 years and free of tumor growth and recurrence, compared to 46 percent of the patients receiving isotretinoin alone.

Dinutuximab is administered as an intravenous infusion. Because of a number of adverse side effects from the drug, including severe pain, FDA is requiring Unituxin to carry a boxed warning that it irritates nerve cells requiring opioid pain killers. Other adverse effects include nerve damage and life-threatening infusion reactions.

United Therapeutics was granted orphan drug status and priority review for dinutuximab that FDA says reduces review times by 4 months. In addition, under a law passed in 2012, United Therapeutics qualified for a rare pediatric disease priority review voucher that the company can apply to a future FDA review. FDA says it’s only the second such voucher granted by the agency.

Read more:

• Thyroid Cancer Drug Approved by FDA
• Lung Cancer Antibody Given FDA Breakthrough Tag
• Personalized Leukemia Immunotherapy Gets 90 Pct Remission
• Personal Leukemia Immunotherapy Given Breakthrough Status
• FDA Grants Experimental Leukemia Drug Orphan Status

*     *     *