RARE Daily

FDA Approves US WorldMed’s Iwilfin for High-Risk Neuroblastoma

December 15, 2023

Rare Daily Staff

The U.S. Food and Drug Administration approved US WorldMeds’ Iwilfin to reduce the risk of relapse for people with high-risk neuroblastoma, the first for pediatric patients with high-risk neuroblastoma.

The approval is for use in adult and pediatric patients who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy.

Some 700 to 800 cases of neuroblastoma are diagnosed in the United States each year, with 90 percent of diagnoses coming before age 5, according to the American Cancer Society. More than 50 percent 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.

Iwilfin, eflornithine 192 mg tablets, is an oral maintenance therapy for high-risk neuroblastoma, indicated to reduce the risk of relapse in adult and pediatric patients who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy.

“The goal for treating these young patients is to prevent relapse, and advancing therapeutic options is critical to this mission. Iwilfin offers new hope and improved outcomes for these vulnerable children,” said Breck Jones, CEO of US WorldMeds.

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 percent compared to 73 percent of patients in the external control group, and 96 percent of patients treated with Iwilfin were alive compared to 84 percent of external control patients. This corresponded to a 52 percent reduction in the risk of relapse and a 68 percent 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 percent to 41 percent and death risk reduction ranged from 71 percent to 55 percent.

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.

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 more than 50 hospitals that offer collaboration through a network of childhood cancer clinical trials.

Photo: Breck Jones, CEO of US WorldMeds

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