While existing neuroblastoma treatments can help patients achieve remission, sustaining that remission has proven tricky. But now, patients and doctors have a new oral option.
This week, the U.S. FDA approved US WorldMeds’ (USWM) Iwilfin—also known as eflornithine—as a new oral maintenance therapy for high-risk neuroblastoma. The drug, cleared in 192 mg tablets, is indicated to cut the risk of relapse in kids and adults with high-risk neuroblastoma who’ve had at least a partial response to prior multiagent, multimodality treatment, USWM said in a press release.
Between 700 and 800 cases of neuroblastoma are diagnosed in the U.S. each year, according to the American Cancer Society. Ninety percent of diagnoses occur before a patient is 5 years old and more than 50% of neuroblastoma cases are classified as high-risk.
Approximately half of children with high-risk neuroblastoma don’t live more than five years past their diagnosis, USWM added. Avoiding relapse is crucial to improving survival rates, the company notes.
Enter Iwilfin, which was approved based on data from a study of the medication as a maintenance therapy in kids with high-risk neuroblastoma following standard of care treatment, including immunotherapy.
At four years following immunotherapy, the event-free survival (EFS) rate for patients on Iwilfin was 84%, compared to 73% of patients in the control cohort. In terms of overall survival (OS), 96% of patients on Iwilfin were alive, compared to 84% in the control over the same stretch of time.
That corresponds to a 52% reduction in the risk of relapse and a 68% reduction in risk of death overall, USWM said.
The company says its medication is generally well-tolerated, with side effects often manageable through dose modifications. The most common side effects patients experienced were hearing loss, inflammation or infection in the middle ear, fever, pneumonia and diarrhea.
I wilfin represents a joint effort between USWM and the Beat Childhood Cancer Research Consortium at Penn State University, which conducted preclinical and clinical research to help advance the drug.
USWM said Iwilfin will be available in the United States in the coming weeks.

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-eflornithine-adult-and-pediatric-patients-high-risk-neuroblastoma