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. 2025;148(4):443-451.
doi: 10.1159/000542294. Epub 2024 Nov 25.

Results of Long-Term Therapy with a Biosimilar of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

Alexander D Kulagin  1 , Vadim V Ptushkin  2 , Elena A Lukina  3 , Igor L Davydkin  4 , Alexander V Korobkin  5 , Tatiana S Konstantinova  6 , Elena Yu Komartseva  7 , Natalia V Minaeva  8 , Tatiana A Mitina  9 , Olesya U Klimova  1 , Evgeniya G Arshanskaya  2 , Vitalii D Latyshev  3 , Oksana A Markova  10 , Eugene V Zuev  10
Affiliations

Results of Long-Term Therapy with a Biosimilar of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

Alexander D Kulagin et al. Acta Haematol. 2025.

Abstract

Introduction: The study aimed to assess the safety, immunogenicity, and efficacy of long-term therapy with biosimilar of eculizumab (Elizaria®) in paroxysmal nocturnal hemoglobinuria (PNH) patients.

Methods: The study included 30 patients with PNH who had completed previous clinical trials. Of these, 25 patients continued receiving the biosimilar product, and 5 patients switched from the originator product Soliris. The maximum duration of follow-up was 104 weeks, during which the investigational product was administered 52 times at a standard dose.

Results: Throughout the study, the levels of lactate dehydrogenase, hemoglobin, reticulocytes, and PNH clone remained stable compared to baseline, regardless of the previous therapy (p > 0.05). There were no significant differences in the number of patients with chronic kidney disease at different visits, as well as in the number of patients who received donor red blood cell and platelet transfusions during the study (p > 0.05). There were 2 cases of adverse reactions reported in 2 patients (6.6%): elevated aspartate aminotransferase (3.3%) and alopecia (3.3%). Immunogenicity analysis showed no significant differences in the frequency of antidrug antibody detection compared to baseline (p > 0.05).

Conclusion: The study findings confirm the long-term efficacy and safety of biosimilar in patients with PNH.

Keywords: Complement system; Eculizumab; Efficacy; Paroxysmal nocturnal hemoglobinuria; Pharmacodynamics; Pharmacokinetics; Safety.

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Conflict of interest statement

Alexander D. Kulagin: Alexion Pharmaceuticals, Inc: consultancy, research funding; JSC GENERIUM: consultancy, honoraria, research funding. Vadim V. Ptushkin: Alexion Pharmaceuticals, Inc: consultancy, research funding; JSC GENERIUM: research funding; Janssen: consultancy; AbbVie: consultancy; Roche: consultancy. Elena A. Lukina: JSC GENERIUM: research funding; Sanofi Genzyme: honoraria, membership on an entity’s Board of Directors or advisory committees; Shire: Honoraria, Membership on an entity’s Board of Directors or advisory committees. Other: travel reimbursement, research funding. Oksana А. Markova: JSC GENERIUM: employment. Eugene V. Zuev: JSC GENERIUM: employment.

Figures

Fig. 1.
Fig. 1.
Minimum product concentration at the end of the dosing interval and membrane attack complex concentration at first study visits. PK population (N = 5).
Fig. 2.
Fig. 2.
Changes in serum LDH activity during the study (median with interquartile range).
See this image and copyright information in PMC

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