Eculizumab for the treatment of hemolytic paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome and refractory myasthenia gravis

Introduction: Complement blockade by administration of eculizumab has led to impressive clinical improvements in patients suffering from complement-mediated diseases such as PNH and aHUS. In addition, the use of the drug is currently investigated for refractory Myasthenia Gravis (rMG). First results are looking promising.Areas covered: We review the characteristics of the agent, its clinical development and efficacy in the three orphan diseases as demonstrated in several pivotal trials. In addition, attempts to individualize dosage and duration of treatment in PNH and aHUS will be discussed. Finally, commercial aspects according to its unique selling proposition as an agent with a complete blocking capacity of the terminal complement cascade will be discussed.Expert opinion: The drug is very effective and gives rise to a significant improvement in terms of survival and quality of life. However, the extremely high price in some countries is a limiting factor and will prevent an easy access to the drug by all patients. Therefore, it would be an improvement if new complement inhibitors under development will be as efficient and safe as eculizumab, but will lead to a significant reduction of the treatment related costs.