USWM, LLC has received FDA approval for IWILFIN™ (eflornithine) 192 mg tablets, a revolutionary oral maintenance therapy designed for high-risk neuroblastoma. The indication for IWILFIN is to decrease the risk of relapse in both adult and pediatric patients with high-risk neuroblastoma who have exhibited at least a partial response to previous multiagent, multimodality therapy, including anti-GD2 immunotherapy.
Neuroblastoma, a challenging childhood cancer, sees 700-800 cases diagnosed annually in the U.S., with 90% of diagnoses occurring before the age of 5. Over half of these cases are classified as high-risk, contributing to a high mortality rate primarily due to the risk of relapse after achieving remission. Existing treatments are effective in achieving remission, but sustaining it is a challenge, and avoiding relapse is critical to enhancing survival rates.
The FDA approval of IWILFIN is grounded in the outcomes of a multi-site, single-arm, externally controlled study involving children with high-risk neuroblastoma who received IWILFIN as maintenance therapy post standard of care treatment, including immunotherapy. Results from the study revealed that IWILFIN significantly improved event-free survival (EFS) and overall survival (OS) in patients with high-risk neuroblastoma. Four years after immunotherapy, EFS for the IWILFIN-treated group was 84%, compared to 73% in the external control group. Additionally, 96% of IWILFIN-treated patients were alive, compared to 84% of external control patients. This translated to a 52% reduction in the risk of relapse and a 68% reduction in the risk of death. Additional analyses confirmed these results, with relapse risk reduction ranging from 57% to 41% and death risk reduction ranging from 71% to 55%.
Administered orally, IWILFIN is taken with or without food twice daily for two years. It is generally well-tolerated, with manageable side effects through dose modifications. Common side effects include hearing loss, otitis media, pyrexia, pneumonia, and diarrhea. Important Safety Information is provided below.
US WorldMeds collaborated with the Beat Childhood Cancer Research Consortium at Penn State University for preclinical and clinical research, fostering the advancement of this crucial therapy. The Consortium, comprising over 50 hospitals, facilitates collaboration through a network of childhood cancer clinical trials.