FDA Clears First High-Risk Neuroblastoma Treatment

The FDA has approved United Therapeutics’ Unituxin in combination with three other drugs for children with neuroblastoma who have seen at least some response to prior combination therapies.

Unituxin (dinutuximab) is the first treatment specifically targeted for patients with high-risk neuroblastoma and provides a treatment option that prolongs survival in children with the diagnosis — an area of high unmet need, according to Richard Pazdur, director of CDER’s Office of Hematology and Oncology. It is given in combination with granulocyte-macrophage colony-stimulating factor, interleukin-2 and 13-cis-retinoic acid.

In a clinical trial of 226 children that compared the Unituxin cocktail to 13-cis-retinoic acid alone, 63 percent were still alive and in remission three years after randomization, compared with 46 patients in the RA arm. A later overall survival analysis revealed that 73 percent of Unituxin patients were still alive versus 58 percent of controls.

Unituxin carries a boxed warning for severe pain caused by nerve irritation, nerve damage and life-threatening reactions. Patients are required to receive intravenous opioids before, during and for two hours after infusion.

United Therapeutics received both priority review and orphan drug designation, shortening the drug’s review time and conferring an extra five years of marketing exclusivity. The FDA also granted the drugmaker a rare pediatric disease priority review voucher, which gives priority review to a future NDA that would not otherwise qualify. — Lena Freund