Bone Marrow Failure Disorders

Randomized Multicenter Noninferiority Phase III Clinical Trial of the First Biosimilar of Eculizumab

A team of researchers from Russia reports updates from the ECU-PNH-III study, a phase 3 randomized multicenter noninferiority study partially reported at the 2019 American Society of Hematology Annual Meeting. The aim was to evaluate efficacy and safety of an eculizumab biosimilar (Elizaria) vs the original product, Soliris (Alexion Pharma GmbH) in patients with PNH. Elizaria and Soliris were administered at 600 mg weekly for 4 weeks at the initial stage in treatment-naive patients, followed by maintenance therapy at 900 mg every 2 weeks. A total of 16 patients were randomized to Elizaria and 16 were randomized to Soliris. The primary endpoint was a comparative assessment of hemolytic activity based on the area under the lactate dehydrogenase (LDH) concentration–time curve during the maintenance therapy. Mean values of LDH concentration–time curve were similar in both treatment groups without statistically significant differences (P  >.05), and no statistically significant differences in secondary endpoints or safety (transfusions, thrombotic complications, breakthrough hemolysis, and quality-of-life-indicators) were observed.

Annals of Hematology