The FDA has Approved the First Oral Drug for Maintenance Therapy of High-Risk Neuroblastoma

Neuroblastoma most commonly occurs in children under the age of five. The disease usually affects areas of the adrenal gland (positioned over the kidneys) or areas in the neck, chest, or close to the spine.

The recent FDA approval of US WorldMeds’ oral drug Iwilfin (eflornithine) moves it into the position of being the first approved oral therapy for high-risk neuroblastoma (HRNB). Iwilfin is designed to reduce the risk of relapse in pediatric and adult patients.

Indeed, Iwilfin has been found to decrease the risk of relapse in people with HRNB according to last week’s announcement. Ornithine decarboxylase (OD) is critical in polyamine synthesis. When the process is suppressed, tumor formation and growth are also suppressed.

Iwilfin inhibits ornithine decarboxylase (OD), thereby preventing OD from performing its intended mission, which is the accumulation of ammonia that rises to toxic levels affecting the CNS (central nervous system).

More About Iwilfin

Iwilfin is the first FDA-approved treatment that has been developed to lessen the risk of a relapse in children or adults who have HRNB. One criterion is that these patients have previously exhibited a partial response to multi-agents that were intended to optimize the treatment of brain disorders.

The drug is available as an oral inhibitor of OD, a critical enzyme that is involved in neoplastic transformation (prevention of tumor growth).

Ornithine transcarbamylase deficiency is the most common of the urea cycle disorders. It is an X-linked genetic disorder that prevents the breakdown and excretion of ammonia. This allows ammonia to accumulate and rise to toxic levels, affecting the central nervous system.

About the FDA’s Approval

The FDA’s approval is partially based on data derived from a single-arm study of children who have HRBN and were initially treated with standard regimens such as immunotherapy. At four years, results showed an 84% survival compared to a 73% external control cohort.

At four years overall survival after Iwilfin therapy was 96% versus 84% in external controls. Therefore, Iwilfin reduced relapse by 52%. In addition, the risk of death was reduced by 68% as reported by US WorldMeds.

Despite the absence of a randomized clinical study supporting Iwilfin’s efficacy, the FDA’s Advisory Committee voted 14 to 6 in favor of approving the drug.

The board was concerned that giving approval with no randomized trial may set a precedent for applications in the future.

Although Iwilfin does not feature a boxed warning on its label, precautions include hepatotoxicity, loss of hearing, myelosuppression, and fetal toxicity.

In conclusion, Breck Jones, CEO of US WorldMeds, termed Iwilfin as being an option that is valued and needed, especially for pediatric patients. He further stated that Iwilfin offers new hope to vulnerable children.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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