Abstract: Objective To provide suggestions to conduct risk management and risk evaluation and mitigation strategy in China by describing ponatinib risk management experience in the United States (US) and European Union (EU). Methods Using Medline and other Internet-based search engines, we systematically reviewed ponatinib withdrawal case due to serious vascular occlusion events and its reintroduction into the market under the Risk Evaluation and Mitigation Strategy (REMS) in US. We also reviewed ponatinib risk assessment in European Union. Results Ponatinib (Iclusig^®), a kinase inhibitor, was approved for positive chronic myeloid leukemia and positive Philadelphia chromosome positive acute lymphoblastic leukemia. Due to the serious adverse reaction, ponatinib was withdrawn in October 2013. Due to its unique pharmacologic efficacy, it was re-introduced to the market in December 2013 under the unique REMS in US. A similar risk management plan was introduced in EU countries. Conclusion The risk management for pharmaceutical products applies to the life cycle of drug development from Investigational New Drug (IND), New Drug Application (NDA), post-marketing, and even to post-withdrawal and reintroduction. The experience of ponatinib risk management in US and EU might shed some light for the future risk management of drug safety in China.

Key words: ponatinib, withdrawal, reintroduction, risk management, risk evaluation and mitigation strategy