Eflornithine hydrochlorideProduct ingredient for Eflornithine

Eflornithine is an irreversible ornithine decarboxylase inhibitor originally developed as a treatment for human African trypanosomiasis.⁶ Further research has also implicated ornithine decarboxylase in other conditions like facial hirsutism and cancer, especially when ornithine decarboxylase is highly upregulated in tumor cells.^3,7 Additionally, ornithine decarboxylase is activated by c-myc or interacts with ras, both very well-known oncogenes, thus increasing the interest in targeting ornithine carboxylase as a potential cancer treatment.⁸

In 1960 and 2000, the FDA approved eflornithine under the brand names ORNIDYL and VANIQUA for the treatment of African trypanosomiasis and hirsutism, respectively, but has since been discontinued.^5,13 Subsequently, on December 14, 2023, the FDA approved eflornithine again but under the brand name IWILFIN as an oral maintenance therapy to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy. This approval is based on positive results obtained from a multi-site, single-arm, externally controlled study of children with high-risk neuroblastoma, where a 52% reduction in the risk of relapse and a 68% reduction in the risk of death were observed.¹²

ChemSpider

390110

ChEBI

4762

ChEMBL

CHEMBL1201037

Wikipedia

Eflornithine