United Therapeutics announced Tuesday that the FDA approved Unituxin (dinutuximab) as part of first-line therapy for paediatric patients with high-risk neuroblastoma. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, noted that "Unituxin marks the first approval for a therapy aimed specifically for the treatment of patients with high-risk neuroblastoma."
Specifically, United Therapeutics said that Unituxin gained approval in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2) and 13-cis-retinoic acid (RA) for the treatment of children with high-risk neuroblastoma who at least partially responded to previous first-line multiagent, multimodality therapy. Unituxin, which has been granted both FDA priority review and orphan drug status, is a chimaeric monoclonal antibody targeting GD2.
The authorisation was supported by data from a study of 226 paediatric patients with high-risk neuroblastoma, in which patients were randomised to treatment with Unituxin and RA or RA alone for six cycles. In the Unituxin arm, patients were also treated with GM-CSF in cycles 1, 3 and 5 and with IL-2 in cycles 2 and 4. The FDA noted that three years after treatment, results showed that 63 percent of patients in the Unituxin arm were alive and free of tumour growth or recurrence, compared to 46 percent of participants treated with RA alone. Meanwhile, in an updated analysis of survival, 73 percent of ptients who received the Unituxin combination were alive compared with 58 percent of those receiving RA alone.
The FDA indicated that Unituxin will carry a boxed warning alerting that the drug 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. The agency added that United Therapeutics' product may also cause other serious side effects such as infections, eye problems, electrolyte abnormalities and bone marrow suppression.
According to the FDA, the approval gives United Therapeutics a rare paediatric disease priority review voucher, which the agency noted confers priority review to a subsequent drug application that would not otherwise qualify for priority review.
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