Eflornithine
- Generic Name
- Eflornithine
- Brand Names
- Vaniqa
- Drug Type
- Small Molecule
- Chemical Formula
- C6H12F2N2O2
- CAS Number
- 70052-12-9
- Unique Ingredient Identifier
- ZQN1G5V6SR
- Background
Eflornithine is a prescription drug indicated in the treatment of facial hirsutism (excessive hair growth). Eflornithine hydrochloride cream for topical application is intended for use in women suffering from facial hirsutism and is sold by Allergan, Inc. under the brand name Vaniqa. Eflornithine for injection against sleeping sickness was manufactured by Sanofi Aventis and sold under the brand name Ornidyl in the USA. It is now discontinued. Eflornithine is on the World Health Organization's List of Essential Medicines.
- Indication
Eflornithine is indicated in the treatment of facial hirsutism (excessive hair growth).
- Associated Conditions
- Meningoencephalitic stage Trypanosoma brucei gambiense infection
- Associated Therapies
- Hair removal therapy
Norgine Seeks EMA Approval for Eflornithine in High-Risk Neuroblastoma
• Norgine has submitted a marketing authorization application to the EMA for eflornithine to treat high-risk neuroblastoma (HRNB).
• The application follows prior submissions in Australia, Switzerland, and the United Kingdom under Project Orbis.
• Eflornithine is intended as an oral maintenance therapy to reduce relapse risk in pediatric and adult HRNB patients.
• FDA approved eflornithine in December 2023 based on trials showing improved event-free and overall survival.
Eflornithine (Iwilfin) Approved for High-Risk Neuroblastoma to Reduce Relapse Risk
• Eflornithine (Iwilfin) is now FDA-approved to reduce the risk of relapse in high-risk neuroblastoma patients who have shown partial response to prior therapies.
• The approval was based on a Phase II trial demonstrating significantly improved event-free survival and overall survival compared to historical controls.
• Eflornithine inhibits polyamine synthesis, targeting the MYCN oncogene, which is crucial in neuroblastoma development and progression.
• Common adverse effects include otitis media, sinusitis, pneumonia, and diarrhea, necessitating careful monitoring and management of patients on eflornithine.
FDA Approves Eflornithine for High-Risk Neuroblastoma to Reduce Relapse Risk
• The FDA has approved eflornithine (Iwilfin) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB).
• The approval is for patients who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy.
• Efficacy was demonstrated in a controlled trial comparing eflornithine to an external control arm, showing improved event-free and overall survival.
• Common adverse events in the eflornithine arm included otitis media, diarrhea, cough, and various infections, alongside some lab abnormalities.
FDA Panel Supports Eflornithine for High-Risk Pediatric Neuroblastoma
• The FDA's Oncologic Drugs Advisory Committee (ODAC) voted 14-6 in favor of eflornithine to reduce relapse risk in pediatric high-risk neuroblastoma patients.
• The vote was based on data from Study 3b, a single-arm trial with an external control arm from the ANBL0032 trial, showing improved event-free survival.
• Eflornithine's safety profile includes manageable toxicities like hearing loss, with dose adjustments potentially mitigating adverse effects.
• While some panel members expressed concerns about using an externally controlled trial, the overall consensus was that the benefits outweigh the risks.
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