FDA approves US WorldMeds’ breakthrough oral drug Iwilfin for high-risk neuroblastoma maintenance therapy

USA — The FDA has approved US WorldMeds’ oral drug eflornithine, now branded as Iwilfin, marking a significant milestone in the field of high-risk neuroblastoma (HRNB) maintenance therapy.

Iwilfin is the first authorized oral maintenance therapy for this indication, offering a promising avenue to reduce the risk of relapse in both adult and pediatric patients.

The recent approval positions Iwilfin as a novel therapy, especially for pediatric patients, as it aims to decrease the risk of relapse in individuals with HRNB who have shown at least a partial response to multiagent, multimodality treatment, including anti-GD2 immunotherapy.

The American Cancer Society reports that there are between 700 and 800 cases of neuroblastoma diagnosed annually in the United States.

A significant majority of these diagnoses, 90%, occur before a patient reaches the age of 5, and over 50% of neuroblastoma cases fall into the high-risk category.

US WorldMeds CEO Breck Jones emphasizes the importance of Iwilfin as a “new and much-needed treatment option” for HRNB, particularly in pediatric cases.

US WorldMeds highlights that nearly half of children diagnosed with high-risk neuroblastoma do not survive beyond five years post-diagnosis.

The company emphasizes the critical importance of preventing relapse as a key factor in enhancing survival rates for individuals affected by this condition.

Iwilfin operates as an orally available inhibitor of ornithine decarboxylase, a key enzyme in neoplastic transformation.

The drug’s label highlights its impact on reducing the expression levels of oncogenic drivers MYCN and LIN28B, thereby promoting cell aging and preventing cancer development.

The FDA’s approval is substantiated by compelling data from a single-arm, externally controlled study involving children with HRNB.

The study, which included standard regimens and immunotherapy, demonstrated an impressive 84% event-free survival at four years compared to 73% in the external control arm.

Notably, overall survival after Iwilfin treatment reached 96% at four years, a significant improvement compared to the 84% observed in external controls.

US WorldMeds reports that Iwilfin reduces the risk of relapse by 52% and the risk of death by 68%.

While the FDA’s Oncologic Drugs Advisory Committee initially had reservations in October 2023, a 14-6 vote in favor of US WorldMeds eventually sealed the approval.

The panel acknowledged the drug’s lower toxicity compared to typical discussions and recognized its efficacy, albeit cautioning about the lack of randomization in the study.

Iwilfin’s label does not carry a boxed warning but does include precautions against myelosuppression, hepatotoxicity, hearing loss, and embryo-fetal toxicity.

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