{"entity":{"id":"eflornithine","kind":"drug","name":"Eflornithine (DFMO)","aka":[],"tldr":"Eflornithine (DFMO) is an old sleeping-sickness drug repurposed as the first oral maintenance therapy for high-risk neuroblastoma, approved in December 2023 to reduce relapse after immunotherapy.","summary":"Approved 13 December 2023 (US WorldMeds) for adults and children with high-risk neuroblastoma who achieved at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. Evidence: single-arm NMTRC003/003B (n=105) compared with an external control from ANBL0032 (EFS HR 0.48, OS HR 0.32), a controversial externally controlled approval. EU applications filed 2025. Two years of twice-daily tablets.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Eflornithine","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Eflornithine"}],"tags":[],"related":[],"cancers":["neuroblastoma","neuroblastoma-high-risk"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["us-worldmeds"],"institutions":[],"pathways":[],"terms":["mycn-amplification"],"trials":["nmtrc003","nct07287917"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Iwilfin","modality":"Small-molecule ornithine decarboxylase inhibitor","mechanism":"Irreversible inhibitor of ornithine decarboxylase (ODC1), a direct MYCN target, depleting polyamines required for neuroblastoma proliferation.","approvals":[{"region":"US","year":2023,"indication":"Maintenance to reduce relapse risk in high-risk neuroblastoma after ≥PR to prior therapy including anti-GD2"}],"mechanismSteps":["DFMO irreversibly inactivates ornithine decarboxylase","Putrescine, spermidine and spermine fall","MYCN-driven translation and proliferation slow","Residual cells are held in check during two years of maintenance"],"dosing":{"route":"Oral","schedule":"Body-surface-area-based dose twice daily for 2 years (e.g. 768 mg twice daily for BSA >1.5 m2)","monitoring":"Hearing (ototoxicity), liver enzymes, counts, growth"},"toxicity":[{"event":"Hearing loss","anyGradePct":27,"note":"NMTRC003/003B; largely pre-existing from platinum"},{"event":"Otitis media","anyGradePct":27},{"event":"Neutropenia","anyGradePct":24},{"event":"Elevated ALT","anyGradePct":33}],"access":[],"regulatoryEvents":[{"date":"2023-12-13","type":"approval","region":"US","note":"First oral maintenance therapy; externally controlled evidence","source":"https://ascopubs.org/doi/10.1200/JCO.24.00546"},{"date":"2025","type":"filing","region":"EU","note":"MAA submitted to EMA, UK, Australia, Switzerland","source":"https://www.onclive.com/view/eu-approval-is-sought-for-eflornithine-in-high-risk-neuroblastoma"}]},"route":"/drugs/eflornithine/","neighbours":{"cancer":[{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"}],"company":[{"id":"us-worldmeds","kind":"company","name":"US WorldMeds","route":"/companies/us-worldmeds/"}],"term":[{"id":"mycn-amplification","kind":"term","name":"MYCN amplification","route":"/terms/mycn-amplification/"}],"trial":[{"id":"nmtrc003","kind":"trial","name":"NMTRC003/003B (DFMO maintenance)","route":"/trials/nmtrc003/"},{"id":"nct01349881","kind":"trial","name":"S0820, Adenoma and Second Primary Prevention Trial","route":"/trials/nct01349881/"},{"id":"nct07287917","kind":"trial","name":"Study of AMXT 1501 and DFMO in Combination With Standard Therapies in Advanced Solid Tumors","route":"/trials/nct07287917/"}],"bottleneck":[{"id":"b-generic-repurposing","kind":"bottleneck","name":"No incentive to repurpose cheap drugs","route":"/bottlenecks/b-generic-repurposing/"},{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"}]}}