Eculizumab opens a new era of treatment for paroxysmal nocturnal hemoglobinuria

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening and debilitating disorder of hematopoiesis. The only curative treatment is allogeneic stem cell transplantation. Other treatments are generally supportive in nature. Recently, eculizumab, as a targeted, disease-modifying treatment, was approved by the US FDA and the European Commission. Eculizumab is a humanized monoclonal antibody that inhibits complement factor C5. It is the first approved drug that specifically inhibits complement. This article presents the major aspects of PNH that are necessary to understand the mechanism of action of eculizumab. Experience from the pilot study and the Phase III pivotal program of eculizumab in PNH will be summarized and the impact of eculizumab on the future treatment of PNH will be discussed.

Keywords::

• complement
• eculizumab
• hemolytic anemia
• paroxysmal nocturnal hemoglobinuria
• thrombosis

Financial & competing interests disclosure

H Schrezenmeier has received lecture fees from Alexion Pharmaceuticals and served on an advisory board for Alexion Pharmaceuticals. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Notes

PNH: Paroxysmal nocturnal hemoglobinuria.