Eflornithine

The Australian Therapeutic Goods Administration has approved IFINWIL® (eflornithine) for treating adults and children with high-risk neuroblastoma who have responded to prior multimodality therapy.

Norgine has entered a definitive agreement to acquire Theravia from Mérieux Equity Partners, adding key rare disease treatments SIKLOS® for sickle cell disease and ORPHACOL® for genetic bile disorders to its portfolio.

• Norgine has submitted a marketing authorization application to the EMA for eflornithine to treat high-risk neuroblastoma (HRNB). • The application follows prior submissions in Australia, Switzerland, and the United Kingdom under Project Orbis. • Eflornithine is intended as an oral maintenance therapy to reduce relapse risk in pediatric and adult HRNB patients. • FDA approved eflornithine in December 2023 based on trials showing improved event-free and overall survival.

Eflornithine (Iwilfin) is now FDA-approved to reduce the risk of relapse in high-risk neuroblastoma patients who have shown partial response to prior therapies.

• Orbus Therapeutics' eflornithine combination with lomustine did not meet the primary endpoint of overall survival in the Phase III STELLAR trial for recurrent IDH mutant astrocytoma. • The combination therapy showed clinically meaningful improvements in overall survival (OS) and progression-free survival (PFS) compared to lomustine alone. • The STELLAR trial involved 343 patients and the combination therapy was well-tolerated, with common adverse events related to myelosuppression and hearing impairment. • Eflornithine, which blocks ornithine decarboxylase, previously received FDA orphan drug and breakthrough designations for anaplastic glioma.