Norgine seeks approval for neuroblastoma drug, addressing most common solid tumour in young children

Pharma New: Although existing treatments for neuroblastoma can initially achieve remission in patients, the challenge lies in sustaining this remission over time. Norgine has submitted applications seeking approval for oral Iwilfin (eflornithine) as a treatment for high-risk neuroblastoma (HRNB).

This milestone represents the first regulatory applications submitted for the orphan drug in Australia, Switzerland, and the UK. The move is part of the Project Orbis initiative, designed to streamline patient access to novel treatments.

Neuroblastoma is the most common solid tumour found in children under five, with most cases diagnosed at around two years of age. Each year in Australia, about 40 children are diagnosed with neuroblastoma and more than 50% of cases are classified as high-risk. Approximately half of children with high-risk neuroblastoma don’t live more than five years past their diagnosis.

Under an exclusive licensing partnership with specialty pharmaceutical company US WorldMeds, Norgine is poised to spearhead the registration and commercialisation activities of eflornithine across Europe, Australia, and New Zealand.

Leveraging data from the investigational arm of Study 3b and a clinical-trial-derived external control arm of Study ANBL0032, at four years following immunotherapy, the event-free survival (EFS) rate for patients on Iwilfin was 84%, compared to 73% of patients in the control cohort. In terms of overall survival (OS), 96% of patients on Iwilfin were alive, compared to 84% in the control over the same stretch of time. That corresponds to a 52% reduction in the risk of relapse and a 68% reduction in risk of death overall.

Dr David Gillen, Norgine chief medical officer, stated “These submissions via Project Orbis represent an important first step in the regulatory process for eflornithine and re-emphasise Norgine’s passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need.”

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