Drugs / Eflornithine
Eflornithine
also known as
DFMO · eflornithine hydrochloride · alpha-difluoromethylornithine · Alpha-Difluromethylornithine · Dfmo hcl · Difluoromethylornithine · Difluoromethylornithine (DFMO) · Difluromethylornithine · +11 more
Small molecule targets ODC1 via inhibition
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed neuroblastoma 3 milestones | |||||
| Filed | EU (EMA) | — | neuroblastoma | 2025-01-07 | “Norgine today announced that it completed its marketing authorisation application filing to European Medicines Agency (EMA) for eflornithine in high-risk neuroblastoma (HRNB).” norgine.com ↗ |
| Filed | Other | — | neuroblastoma | 2024-04-10 | “marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland” norgine.com ↗ “marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia” norgine.com ↗ |
| Filed | UK (MHRA) | — | neuroblastoma | 2024-04-10 | “marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia,...” norgine.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Vaniqa | — | 2001-03-19 | europa.eu ↗ |
| Approved | US (FDA) | ORNIDYL | — | 1990-11-28 | fda.gov ↗ |
Trials 38
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT05717153 | Oct 2023 → Jan 2027 expected | malignant glioma | Mayo Clinic | Recruiting | No outcome recorded |
| Phase 13 trials | ||||||
| Phase 1 | NCT05879367 | Jul 2023 → Jun 2026 overdue | IDH-wildtype glioblastoma, astrocytoma (excluding glioblastoma) | Orbus Therapeutics, Inc. | Recruiting | No outcome recorded |
| Phase 1 | NCT02030964 | Jan 2014 → Dec 2023 | neuroblastoma | New Approaches to Neuroblastoma Therapy Consortium | Completed | No outcome recorded |
| Phase 1 | NCT01059071 | Feb 2010 → Oct 2014 | neuroblastoma | Giselle Sholler | Completed | No outcome recorded |
| Phase 1/25 trials | ||||||
| Phase 1/2 | NCT07468136 | Jun 2026 → Oct 2030 expected | IDH-wildtype glioblastoma, anaplastic oligodendroglioma, astrocytoma, IDH-mutant, grade 3, astrocytoma, IDH-mutant, grade 4 +1 | Mayo Clinic | Recruiting | No outcome recorded |
| Phase 1/2 | NCT06465199 | May 2026 → May 2033 expected | Ewing sarcoma, atypical teratoid rhabdoid tumor, diffuse intrinsic pontine glioma, embryonal tumor with multilayered rosettes +2 | Milton S. Hershey Medical Center | Recruiting | No outcome recorded |
| Phase 1/2 | NCT07287917 | Jan 2026 → Feb 2028 expected | cutaneous melanoma, hormone receptor-positive breast cancer | Aminex Therapeutics, Inc. | Recruiting | No outcome recorded |
| Phase 1/2 | NCT06892678 | Apr 2025 → Apr 2030 expected | Ewing sarcoma, osteosarcoma | Montefiore Medical Center | Recruiting | No outcome recorded |
| Phase 1/2 | NCT02139397 | Jun 2014 → Jan 2024 | neuroblastoma | Giselle Sholler | Completed | No outcome recorded |
| Phase 214 trials · 1 met primary · 2 missed | ||||||
| Phase 2 | NCT07321912 | Jun 2026 → Jun 2031 expected | Ewing sarcoma, osteosarcoma | Milton S. Hershey Medical Center | Recruiting | No outcome recorded |
| Phase 2 | NCT06059118 | Oct 2023 → Jan 2027 expected | prostate cancer | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Recruiting | No outcome recorded |
| Phase 2 | NCT04301843 | Sep 2020 → Oct 2028 expected | neuroblastoma | Giselle Sholler | Active not recruiting | No outcome recorded |
| Phase 2 | NCT03794349 | Jul 2019 → Mar 2025 overdue | neuroblastoma | Children's Oncology Group | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02395666 | Mar 2015 → Mar 2018 | neuroblastoma | Giselle Sholler | Completed | Met primary |
| Phase 2 | NCT00983580 | Aug 2009 → Oct 2016 | adenomatous colon polyp | National Cancer Institute (NCI) | Completed | Missed primary |
| Phase 2 | NCT00601640 | Jan 2007 → Feb 2010 | benign neoplasm of skin | University of Arizona | Completed | No outcome recorded |
| Phase 2 | NCT00033371 | Dec 2001 → Feb 2009 | classic or attenuated familial adenomatous polyposis, colorectal cancer | National Cancer Institute (NCI) | Completed | Missed primary |
| Phase 2 | NCT00086736 | Nov 2001 → Nov 2003 | prostate cancer | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 2 | NCT00021294 | May 2001 → Jun 2002 | precancerous condition, skin cancer | University of Arizona | Completed | No outcome recorded |
| Phase 2 | NCT00176995 | Mar 1999 → Oct 2000 | Pseudofolliculitis barbae | Hordinsky, Maria K., MD | Completed | No outcome recorded |
| Phase 2 | NCT00006101 | Oct 1998 → Dec 2012 | prostate cancer | Thomas E. Ahlering | Completed | No outcome recorded |
| Phase 2 | NCT00006079 | Jun 1998 → Apr 2004 | cervical cancer, dysplasia of cervix | M.D. Anderson Cancer Center | Completed | No outcome recorded |
| Phase 2 | NCT00003076 | Oct 1995 → Jun 2002 | esophageal cancer | University of Michigan Rogel Cancer Center | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT00489658 | Oct 2002 → ? | human African trypanosomiasis | Epicentre | Terminated | No outcome recorded Stop: Enrollment |
| Phase 39 trials · 1 met primary · 1 missed | ||||||
| Phase 3 | NCT02796261 | Jul 2016 → Jun 2023 | anaplastic astrocytoma | Orbus Therapeutics, Inc. | Unknown | No outcome recorded |
| Phase 3 | NCT01483144 | Oct 2013 → Nov 2018 | classic familial adenomatous polyposis | Cancer Prevention Pharmaceuticals, Inc. | Completed | Missed primary |
| Phase 3 | NCT01349881 | Aug 2013 → Sep 2027 expected | colorectal neoplasm | SWOG Cancer Research Network | Active not recruiting | No outcome recorded |
| Phase 3 | NCT01245816 | Mar 2011 → May 2013 | classic familial adenomatous polyposis | Cancer Prevention Pharmaceuticals, Inc. | Withdrawn | No outcome recorded |
| Phase 3 | NCT00330148 | Mar 2001 → Jun 2004 | human African trypanosomiasis | Epicentre | Terminated | No outcome recorded |
| Phase 3 | NCT00003814 | Feb 1999 → May 2007 | urinary bladder cancer | National Cancer Institute (NCI) | Completed | No outcome recorded |
| Phase 3 | NCT00118365 | Jul 1998 → Aug 2008 | precancerous condition | National Cancer Institute (NCI) | Completed | No outcome recorded |
| Phase 3 | NCT00005884 | ? → Dec 2000 | skin carcinoma | University of Wisconsin, Madison | Completed | No outcome recorded |
| Phase 3 | NCT00146627 | ? → Jun 2008 | human African trypanosomiasis | Drugs for Neglected Diseases | Completed | Met primary |
| Phase 44 trials | ||||||
| Phase 4 | NCT06976424 | Jul → Dec 2025 overdue | disorder of pilosebaceous unit | Khyber Teaching Hospital | Recruiting | No outcome recorded |
| Phase 4 | NCT01817894 | Jan 2012 → Oct 2013 | hypertrichosis | Bispebjerg Hospital | Completed | No outcome recorded |
| Phase 4 | NCT00906880 | Apr 2009 → Sep 2010 | human African trypanosomiasis | Drugs for Neglected Diseases | Completed | No outcome recorded |
| Phase 4 | NCT00152048 | Nov 2004 → Oct 2005 | hypertrichosis | Allergan | Completed | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT03581240 | — | embryonal tumor with multilayered rosettes, ependymoblastoma, medulloblastoma, medulloepithelioma +2 | Giselle Sholler | Available | No outcome recorded |
| Comparator or background therapy6 trials | ||||||
| Phase 2 | NCT06957834 Comparator | Jun 2023 → Jul 2024 | hyperpigmentation of the skin | National Skin Centre | Completed | No outcome recorded |
| Phase 2 | NCT04091022 Comparator | Jan 2021 → Dec 2025 | skin cancer | University of Alabama at Birmingham | Completed | No outcome recorded |
| Phase 2 | NCT02794428 Comparator | Sep 2016 → Dec 2022 | gastric cancer, gastric intestinal metaplasia | Vanderbilt-Ingram Cancer Center | Completed | No outcome recorded |
| Phase 2 | NCT02559778 Comparator | Sep 2015 → Sep 2030 expected | neuroblastoma | Giselle Sholler | Recruiting | No outcome recorded |
| Phase 2/3 | NCT01685827 Comparator | Oct 2012 → Nov 2016 | human African trypanosomiasis | Drugs for Neglected Diseases | Completed | No outcome recorded |
| Phase 4 | NCT04403282 Comparator | Sep 2020 → Jun 2023 | Pseudofolliculitis barbae | Walter Reed National Military Medical Center | Terminated | No outcome recorded |
News releases announcing trial results or a regulatory action · 10
| Date | Issuer | Release |
|---|---|---|
| 2025-07-31 | Norgine | Regulatory FOR AUSTRALIAN MEDIA ONLYNorgine welcomes PBS listing in Australia of IFINWIL® (eflornithine) for patients with high-risk neuroblastoma (HRNB) norgine.com ↗
Eflornithine is now reimbursed by the government under Australia’s Pharmaceutical Benefits Scheme (PBS). |
| 2025-07-08 | Norgine | Regulatory Swissmedic approves IFINWIL® (eflornithine) for children diagnosed with high-risk neuroblastoma (HRNB) norgine.com ↗
Norgine, a leading European specialty pharmaceutical company, is pleased to announce that Swissmedic has approved the registration of IFINWIL® (eflornithine) as monotherapy for the treatment of paediatric patients from one year with high-risk neuroblastoma (HRNB)1. |
| 2025-04-17 | Norgine | Regulatory Norgine welcomes TGA registration in Australia of IFINWIL® (eflornithine) for adults and children diagnosed with high-risk neuroblastoma (HRNB)1 norgine.com ↗
Norgine is pleased to announce that the Australian Therapeutic Goods Administration (TGA) has approved the registration of IFINWIL® (eflornithine) for the treatment of adults and paediatric patients with high-risk neuroblastoma (HRNB), who have responded to prior multiagent, multimodality therapy. |
| 2025-01-07 | Norgine | Regulatory Norgine submits Marketing Authorisation Application to the European Medicines Agency for eflornithine (difluoromethylornithine [DFMO]) in high-risk neuroblastoma norgine.com ↗
Norgine today announced that it completed its marketing authorisation application filing to European Medicines Agency (EMA) for eflornithine in high-risk neuroblastoma (HRNB). |
| 2024-04-15 | Norgine | Regulatory Norgine B.V. submits Marketing Authorisation Application via Project Orbis for eflornithine (difluoromethylornithine [DFMO]) in high-risk neuroblastoma norgine.com ↗
Norgine B.V. today announced its first marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. |
| 2023-12-14 | USWM, LLC (dba US WorldMeds) | Regulatory US WorldMeds Announces FDA Approval of IWILFIN™ (eflornithine) to Strengthen Fight Against Aggressive Childhood Cancer usworldmeds.com ↗
USWM, LLC (US WorldMeds) today announced that the U.S. Food and Drug Administration (FDA) has approved IWILFIN™ (eflornithine) 192 mg tablets, a groundbreaking oral maintenance therapy for high-risk neuroblastoma. |
| 2017-11-03 | Drugs for Neglected Diseases | Results Phase II/III studies show high efficacy and safety of fexinidazole, the first oral treatment for sleeping sickness | DNDi dndi.org ↗
Treatment success rates of 91.2% were measured for fexinidazole, with 97.6% for NECT, 18 months after the end of treatment. |
| 2013-07-10 | Drugs for Neglected Diseases | Regulatory Three Neglected-Disease Treatments Newly Added to WHO Essential Medicines List for Paediatric Use | DNDi dndi.org ↗
Nifurtimox-eflornithine combination therapy (NECT) was added to the EMLc for the treatment of late-stage sleeping sickness (human African trypanosomiasis) in children. |
| 2009-05-14 | Drugs for Neglected Diseases | Regulatory NECT Added to WHO Essential Medicines List as Combination Treatment Against Sleeping Sickness | DNDi dndi.org ↗
According to the WHO, NECT can now be used in patients and will provide an opportunity to improve the management of sleeping sickness cases. |
| 2008-12-08 | Drugs for Neglected Diseases | Results Positive Results for Improved Treatment Against Sleeping Sickness | DNDi dndi.org ↗
Pivotal Phase III clinical trial results conclusively show that Nifurtimox-Eflornithine Combination Therapy (NECT) is well tolerated and effective against the advanced stage of T. b. gambiense sleeping sickness, a fatal disease that threatens 60 million people in sub-Saharan Africa. |
All press releases naming this drug 11 releases
Evidence & citations 24 cited values
Every value below carries the sentence it was read from. 46 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | eflornithine | “Solaraze-topical diclofenac 3% and Vaniqa-topical eflornithine hydrochloride Cream, 13.9% applied to bilateral arms for a period of 9 months” NCT04091022 ↗33“co-administration of nifurtimox (10 days, 15 mg/kg/day, p.o. TID) and eflornithine (7 days, 400 mg/kg/day, i.v. BID)” NCT00906880 ↗ |
| Known as | alpha-difluoromethylornithine | “A clinical study of the efficacy of oral alpha-difluoromethylornithine (eflornithine or DFMO) in male and female subjects ages 30-60 with gastric premalignant lesions in two...” NCT02794428 ↗ |
| Known as | Alpha-Difluromethylornithine | “A Randomized Double-Blind Study of Alpha-Difluromethylornithine (DFMO) Versus Placebo in Patients With Cervical Intraepithelial Neoplasia (CIN) Grade 2-3” NCT00006079 ↗ |
| Known as | DFMO | “Determine whether oral eflornithine (DFMO) given in this study will cause significant reduction of the Ki67 labelling index in subjects with intestinal type Barrett's esophagus...” NCT00003076 ↗13“A clinical study of the efficacy of oral alpha-difluoromethylornithine (eflornithine or DFMO) in male and female subjects ages 30-60 with gastric premalignant lesions in two...” NCT02794428 ↗ “A Randomized Double-Blind Study of Alpha-Difluromethylornithine (DFMO) Versus Placebo in Patients With Cervical Intraepithelial Neoplasia (CIN) Grade 2-3” NCT00006079 ↗ “Patients receive oral eflornithine (DFMO) daily.” NCT00005884 ↗ “Eflornithine (DFMO)” NCT06465199 ↗ “eflornithine (DFMO)” NCT00021294 ↗ |
| Known as | Dfmo hcl | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | Difluoromethylornithine | “Topical Difluoromethylornithine (DFMO) With and Without a Topical Corticosteroid Cream (Triamcinolone 0.1%)” NCT00021294 ↗ |
| Known as | Difluoromethylornithine (DFMO) | “Difluoromethylornithine (DFMO)” NCT00006101 ↗ |
| Known as | Difluromethylornithine | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | eflornithine (Ornidyl) | “eflornithine (Ornidyl)” NCT00906880 ↗ |
| Known as | Eflornithine HCL | “Eflornithine HCL ointment” NCT00601640 ↗ |
| Known as | eflornithine hydrochloride | “Solaraze-topical diclofenac 3% and Vaniqa-topical eflornithine hydrochloride Cream, 13.9% applied to bilateral arms for a period of 9 months” NCT04091022 ↗ |
| Known as | Eflornithine hydrochloride monohydrate | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | Eflornitina | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | Iwilfin | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | MDL 71,782 A | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | NSC-270295 | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | NSC-337250 | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | Ornidyl | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | RMI-71782-A | ChEMBL registry synonym — accepted as the source's own label CHEMBL830 ↗ |
| Known as | Vaniqa | “Solaraze-topical diclofenac 3% and Vaniqa-topical eflornithine hydrochloride Cream, 13.9% applied to bilateral arms for a period of 9 months” NCT04091022 ↗ |
| Action | Inhibit | “Difluoromethylornithine (DFMO) is an antiproliferative agent, inhibiting ornithine decarboxylase” PMID 18268112 ↗ Feb 20081“Ornithine decarboxylase inhibitor” CHEMBL830 ↗ |
| Modality | Small molecule | — CHEMBL830 ↗ |
| Route | Oral | “oral eflornithine (DFMO)” NCT00003076 ↗ |
| Target | ODC1 | “Ornithine decarboxylase inhibitor” CHEMBL830 ↗ |