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논문분류	춘계학술대회 초록집
제목	Totality of the Evidence Supports Extrapolation of the Atypical Hemolytic Uremic Syndrome Indication for EPYSQLI, Biosimilar to Reference Eculizumab
저자	Jihyun Han
출판정보	2025; 2025(1):
키워드	aHUS, Atypical Hemolytic Uremic Syndrome , Eculizumab, Biosimilar, Extrapolation
초록	Eculizumab, a complement 5 (C5) inhibitor of terminal pathway, has revolutionized the management of rare diseases such as paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and neuromyelitis optica spectrum disorder (NMOSD) worldwide including Korea. The biosimilar of eculizumab, EPYSQLI® (SB12), has been approved in Europe, United States, and Korea. We performed an analysis to extrapolate the indications of aHUS from PNH data, based on the ‘totality-of-the-evidence’ demonstrating biosimilarity of SB12 to Soliris®, the eculizumab reference product (ECU-RP). Extensive analytical (more than 40 assays) and non-clinical assessments were performed comparing SB12 to ECU-RP. Clinical studies were conducted for comparing SB12 to ECU-RP in terms of pharmacokinetics (PK), pharmacodynamics (PD), efficacy, safety, tolerability, and immunogenicity in healthy subjects (phase I) and patients with complement inhibitor-naïve PNH (phase III) in 8 countries including Asian countries. Structural, physicochemical, and biological characterization results demonstrated that SB12 is highly similar to ECU-RP. Mechanism of action-related biological activities were similar in respect to overall quality attributes (e.g., potency binding activity). Equivalent PK and efficacy with comparable safety, PD, and immunogenicity between SB12 and ECU-RP were found from healthy subjects and naïve PNH patients through phase I and III trials. Based on the totality-of-the-evidence, the Korean Ministry of Food and Drug Safety approved EPYSQLI® for the use in aHUS by principle of extrapolation in January 2024, together with PNH.
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