Unknown MAH — Phelinun EMA EPAR public assessment report (EMEA/H/C/005173, 2020)

Report summary

This report details the marketing authorization application for Phelinun (melphalan) in the EU, a hybrid medicinal product referencing Alkeran. The application seeks approval for melphalan in various oncology indications, including a new therapeutic indication for conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (HSCT) in haematological diseases for both adult and pediatric populations. The report assesses Phelinun's quality, non-clinical, and clinical aspects, including its genotoxic nature, pharmacokinetic properties, and environmental risk. Efficacy and safety data, primarily from literature-based studies, are presented for melphalan-containing conditioning regimens (e.g., FM, BuCyMel) compared to busulfan-containing regimens. While acknowledging methodological limitations like lack of randomization, the report highlights melphalan's established efficacy and safety profile, including common adverse drug reactions, and its role in reducing relapse risk and improving survival in certain patient cohorts, despite some increased relapse risk in specific high-risk groups.

Key findings — cited to the page