Iwilfin FDA Approval History
Last updated by Judith Stewart, BPharm on Dec 15, 2023.
FDA Approved: Yes (First approved December 13, 2023)
Brand name: Iwilfin
Generic name: eflornithine
Dosage form: Tablets
Company: US WorldMeds, LLC
Treatment for: Neuroblastoma
Iwilfin (eflornithine) is an ornithine decarboxylase inhibitor used to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB).
- Iwilfin is indicated to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy.
- High-risk neuroblastoma is a highly aggressive solid tumor that most commonly presents in early childhood.
- Iwilfin contains eflornithine, which works as an irreversible inhibitor of the enzyme ornithine decarboxylase. Ornithine decarboxylase plays a critical role in polyamine synthesis and a transcriptional target of MYCN, and inhibiting this process suppresses tumor growth and formation.
- FDA approval of Iwilfin was 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.
- Iwilfin tablets are administered orally twice daily with or without food until disease progression, unacceptable toxicity, or for a maximum of two years.
- Warnings and precautions associated with Iwilfin include myelosuppression, hepatotoxicity, hearing loss, and embryo-fetal toxicity.
- Common adverse reactions include hearing loss, otitis media, pyrexia, pneumonia, and diarrhea. Common Grade 3 or 4 laboratory abnormalities include increased ALT, increased AST, decreased neutrophil count, and decreased hemoglobin.
- Eflornithine was first approved under the brand name Ornidyl (eflornithine hydrochloride) in 1990 in injection form for the treatment of African trypanosomiasis (sleeping sickness). A topical cream formulation of eflornithine was approved under the brand name Vaniqa (eflornithine hydrochloride) in 2000 for the treatment of hirsutism (unwanted facial hair) in women. Both Ornidyl and Vaniqa are now discontinued in the United States.
Development timeline for Iwilfin
| Date | Article |
|---|---|
| Dec 14, 2023 | Approval FDA Approves Iwilfin (eflornithine) as Maintenance Therapy for High-Risk Neuroblastoma |
Further information
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