DrOmics Labs

FDA Approves First Oral High-Risk Neuroblastoma Maintenance Therapy

The U.S. Food and Drug Administration (FDA) has recently approved the first and only oral maintenance therapy, Iwilfin (eflornithine), to strengthen the fight against aggressive childhood cancer, specifically high-risk neuroblastoma. This approval marks a significant advancement in the treatment of this condition, providing a new and much-needed treatment option for both adult and paediatric patients.

What is Neuroblastoma?

Neuroblastoma is a type of cancer that develops from immature nerve cells found in several areas of the body, most commonly in the adrenal glands, neck, chest, or spinal cord. It is the most common cancer in infants and the third most common cancer in children, accounting for 6% of all childhood cancers. High-risk neuroblastoma is a particularly aggressive form of the disease that is difficult to treat and has a high rate of relapse.

What is Iwilfin?

Iwilfin (eflornithine) is a groundbreaking oral maintenance therapy for high-risk neuroblastoma. It is indicated to reduce the risk of relapse in adult and paediatric patients who have undergone multimodality treatment, including anti-GD2 immunotherapy. Iwilfin is the first authorized oral maintenance therapy for this indication, according to the announcements. US WorldMeds CEO Breck Jones in a statement called Iwilfin a “new and much-needed treatment option” particularly for paediatric patients.

How Does Iwilfin Work?

Iwilfin works by inhibiting the activity of an enzyme called ornithine decarboxylase (ODC), which is involved in the production of polyamines. Polyamines are essential for cell growth and division, and cancer cells have been shown to have higher levels of polyamines than normal cells. By inhibiting ODC, Iwilfin reduces the levels of polyamines in cancer cells, which slows down their growth and division.

 Clinical Trials and Efficacy

The FDA approval of Iwilfin was based on the results of a phase III clinical trial that involved 564 patients with high-risk neuroblastoma who had undergone multimodality treatment, including anti-GD2 immunotherapy. The trial showed that patients who received Iwilfin had a significantly lower risk of relapse compared to those who received placebo. The estimated two-year event-free survival rate was 66% in the Iwilfin group and 48% in the placebo group.

Dosage and Administration

Iwilfin is available in 192 mg tablets and is taken orally twice daily. The recommended duration of treatment is two years, but it may be extended based on the patient’s response to treatment and the risk of relapse.

Side Effects and Precautions

The most common side effects of Iwilfin include bone marrow suppression, liver toxicity, and hearing loss. Patients taking Iwilfin should be monitored regularly for these side effects, and the dosage may need to be adjusted or treatment discontinued if they occur. Iwilfin’s label does not come with a boxed warning but carries precautions against myelosuppression, hepatotoxicity, and ototoxicity.


The approval of Iwilfin marks a significant advancement in the treatment of high-risk neuroblastoma, providing a new and much-needed treatment option for both adult and paediatric patients. The drug’s efficacy in reducing the risk of relapse in patients who have undergone multimodality treatment, including anti-GD2 immunotherapy, has been demonstrated in a phase III clinical trial. While Iwilfin has some side effects, it is an important addition to the treatment options available for high-risk neuroblastoma.





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