United Therapeutics on Monday announced that the European Commission cleared Unituxin (dinutuximab) for the treatment of high-risk neuroblastoma in patients aged 12 months to 17 years who were previously administered induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and autologous stem cell transplantation. Approval of the therapy was supported by clinical data from the ANBL0032 trial comparing Unituxin in combination with 13-cis-retinoic acid (RA) to RA alone. The monoclonal antibody is administered in combination with granulocyte-macrophage colony-stimulating factor, interleukin-2 and isotretinoin.
In the study, 226 paediatric patients with high-risk neuroblastoma were randomised to six cycles of treatment with Unituxin plus RA or RA alone. In the combination arm, patients received Unituxin plus granulocyte macrophage-colony stimulating factor and RA in cycles 1, 3 and 5, Unituxin plus interleukin-2 and RA in cycles 2 and 4 and RA in cycle 6. The primary endpoint of the study was investigator-assessed event-free survival (EFS), while secondary endpoints included overall survival (OS).
In the primary intent-to-treat analysis, the two-year estimates of EFS were 66 percent among patients receiving dinutuximab immunotherapy plus isotretinoin, versus 48 percent in patients administered isotretinoin alone. Meanwhile, the three-year OS rates were 80 percent and 67 percent in the combination and monotherapy arms, respectively, while the five-year OS rate was 74 percent for Unituxin-treated patients, compared to 57 percent for the monotherapy arm.
In March, Unituxin was approved by the FDA for the treatment of paediatric patients with high-risk neuroblastoma, marking the first approval of paediatric patients with high-risk neuroblastoma who experienced at least a partial response to prior first-line multi-agent multi-modality therapy.
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