USWM, LLC announced that the FDA approved IWILFIN™ (eflornithine) 192 mg tablets, a groundbreaking oral maintenance therapy for high-risk neuroblastoma. IWILFIN is indicated to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy.
According to the American Cancer Society, 700-800 cases of neuroblastoma are diagnosed in the U.S. each year, with 90% of diagnoses coming before age 5. Over 50% of these cases are classified as high-risk. High-risk neuroblastoma is a challenging disease, with a high mortality rate driven primarily by the risk of relapse after achieving remission. Approximately half of children with high-risk neuroblastoma do not survive beyond five years from diagnosis. Although existing treatments are effective in helping patients achieve remission, patients lack options to sustain it. Avoiding relapse is crucial to improving survival rates.
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 IWILFIN-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%.
IWILFIN is taken orally, with or without food, twice daily for two years. IWILFIN is generally well-tolerated, with side effects typically manageable through dose modifications. The most common side effects are hearing loss, otitis media, pyrexia, pneumonia, and diarrhea. Important Safety Information can be found below.
US WorldMeds partnered with the Beat Childhood Cancer Research Consortium at Penn State University, which conducted the preclinical and clinical research to help advance this vital therapy. The Consortium represents a group of over 50 hospitals that offer collaboration through a network of childhood cancer clinical trials.
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