FDA approves Iwilfin (eflornithine) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma – US World Meds

The FDA has approved eflornithine (Iwilfin, USWM, LLC) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy.

This represents the first FDA approval of a therapy intended to reduce the risk of relapse in pediatric patients with HRNB.

The approval of Iwilfin is based on the results of a multi-site, single-arm, externally controlled study of children with high-risk neuroblastoma who received Iwilfin as maintenance therapy following standard of care treatment, including immunotherapy.

The study demonstrated that the addition of Iwilfin improved event-free survival (EFS) and overall survival (OS) in patients with high-risk neuroblastoma. At four years following immunotherapy, EFS in the Iwif in- treated patient group was 84% compared to 73% of patients in the external control group, and 96% of patients treated with Iwilfin were alive compared to 84% of external control patients. This corresponded to a 52% reduction in the risk of relapse and a 68% reduction in the risk of death. Across additional analyses to confirm the results of the externally controlled study design, the relapse risk reduction ranged from 57% to 41% and death risk reduction ranged from 71% to 55%.