US FDA approves drug that prolongs survival in rare paediatric cancer

The US Food and Drug Administration (FDA) has approved dinutuximab (United Therapeutics’ Unituxin) as part of first-line therapy for patients with a high-risk version of the rare childhood cancer, neuroblastoma. The drug is the first medicine to be licensed specifically for this condition, which affects approximately one out of 100,000 children. 

Neuroblastoma is a cancer of immature nerve cells called neuroblasts and it usually occurs in children under the age of five. At the moment, treatment of neuroblastoma usually involves surgery, chemotherapy, radiation therapy and sometimes bone marrow transplant. Even if the cancer is eliminated through treatment, it is common for it to recur. Dinutuximab has received approval for treatment of high-risk neuroblastoma after patients have completely or partially responded to initial multi-pronged therapy. 

The FDA says approval of the drug “fulfils a critical need by providing a treatment option that prolongs survival”. United Therapeutics was given a rare paediatric disease priority review voucher, which means a subsequent medicine submitted for approval can receive priority for review. 

The most common side effects associated with dinutuximab include severe pain, which needs to be treated with narcotic painkillers, fever, low platelet counts and infusion reactions.

A spokesperson for the European Medicines Agency says the product is under evaluation as an orphan medicine. 

• This article was amended on 16 March 2015 to clarify that dinutuximab is approved for use first-line only in a subset of patients with high-risk neuroblastoma.