Antiparasitic Wins FDA Approval for High-Risk Neuroblastoma

The US Food and Drug Administration (FDA) approved eflornithine (Iwilfin) to reduce the risk for relapse in 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 the first of a therapy intended to reduce the risk for relapse in pediatric patients with high-risk neuroblastoma, the agency said in a press release. 

Developed in the 1970s, an injectable formulation of eflornithine has been used as an antiparasitic to treat sleeping sickness in Africa, and a topical version has been used to curb excessive facial hair growth in women. These two formulations of eflornithine, however, have been discontinued in the United States. 

The new approval is for an oral formulation dosed according to body surface area. Studies in more than 100 patients and more than 1000 controls demonstrated a reduction in event-free survival, defined as disease progression, relapse, secondary cancer, or death from any cause.

Findings from the analysis revealed a 51%-57% decrease in event-free survival in patients who had at least a partial response to previous treatment. Eflornithine was also associated with improved overall survival (hazard ratio, 0.29-0.45). 

Adverse events in 5% of patients or more include otitis media, diarrhea, cough, sinusitis, pneumonia, upper respiratory tract infection, conjunctivitis, vomiting, pyrexia, allergic rhinitis, decreased neutrophils, increased ALT, increased AST, hearing loss, skin infection, and urinary tract infection. 

M. Alexander Otto is a physician assistant with a master’s degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: [email protected].

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