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Vaniqa (EFLORNITHINE)
Eflornithine inhibits ornithine decarboxylase, reducing polyamine synthesis and affecting cancer cell growth and metabolism.
Vaniqa (Eflornithine) is a small molecule antiprotozoal drug originally developed by Schering-Plough and currently owned by Uswm. It targets ornithine decarboxylase, an enzyme involved in the growth and proliferation of certain parasites. Vaniqa is FDA-approved for the treatment of African trypanosomiasis and female facial hirsutism. The drug is off-patent, with no active Orange Book patents, and has a bioavailability of 55%. Key safety considerations include its potential to cause skin irritation and allergic reactions.
At a glance
| Generic name | EFLORNITHINE |
|---|---|
| Sponsor | Uswm |
| Drug class | Antiprotozoal |
| Target | Ornithine decarboxylase (ODC) |
| Modality | Small molecule |
| Therapeutic area | Other |
| Phase | FDA-approved |
| First approval | 1990 |
Mechanism of action
Eflornithine works by blocking the enzyme ornithine decarboxylase, which is crucial for making polyamines. Polyamines are important for cell growth and cancer development. By reducing polyamines, eflornithine can slow down or stop cancer cells from growing and spreading.
Approved indications
- African trypanosomiasis
- Female Facial Hirsutism
Common side effects
- Acne
- Pseudofolliculitis Barbae
- Stinging Skin
- Headache
- Burning Skin
- Dry Skin
- Pruritus (itching)
- Erythema (redness)
- Tingling Skin
- Dyspepsia
- skin irritation
- rash
Key clinical trials
- Study of AMXT 1501 and DFMO in Combination With Standard Therapies in Advanced Solid Tumors (PHASE1,PHASE2)
- Retifanlimab With or Without Difluoromethylornithine for the Treatment of Progressive High Grade Gliomas (PHASE1,PHASE2)
- Targeting ODC as an Immunotherapeutic Target in STK11 (LKB1) Pathway-Deficient NSCLC (DFMO) (PHASE1,PHASE2)
- DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma (PHASE2)
- Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas (PHASE1,PHASE2)
- An Intermediate Expanded Use Trial of DFMO
- Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PHASE2)
- Irinotecan Hydrochloride, Temozolomide, and Dinutuximab With or Without Eflornithine in Treating Patients With Relapsed or Refractory Neuroblastoma (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Vaniqa CI brief — competitive landscape report
- Vaniqa updates RSS · CI watch RSS