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Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD
ABP 959 is being developed as a biosimilar to Soliris® (eculizumab) reference product (RP), which was approved under orphan designation for a group of rare diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), an...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Springer Berlin Heidelberg
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10640484/ https://www.ncbi.nlm.nih.gov/pubmed/37817009 http://dx.doi.org/10.1007/s00277-023-05439-4 |
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author | McBride, Helen J. Frazer-Abel, Ashley Thiemann, Sandra Lehto, Sonya G. Hutterer, Katariina M. Liu, Jennifer |
author_facet | McBride, Helen J. Frazer-Abel, Ashley Thiemann, Sandra Lehto, Sonya G. Hutterer, Katariina M. Liu, Jennifer |
author_sort | McBride, Helen J. |
collection | PubMed |
description | ABP 959 is being developed as a biosimilar to Soliris® (eculizumab) reference product (RP), which was approved under orphan designation for a group of rare diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). Development of biosimilars for therapeutics approved for rare disease indications must provide scientific rationale based on the totality of evidence (TOE). To support the TOE and the scientific justification for extrapolation to all approved indications for eculizumab RP, including but not limited to aHUS and NMOSD, we utilized simulated ex-vivo pharmacodynamic (PD) assessments to compare the complement component 5 (C5) inhibitory activity of ABP 959 and the RP. Hemolysis activity of CH50 and AH50, and Wieslab CP, AP, and LP endpoints represent the three complement activation pathways (classical, alternative, and lectin), all of which share the terminal pathway and require C5 for activity. These endpoints were evaluated in normal serum, simulated aHUS serum, and simulated NMOSD serum to provide a robust comparison. The results support the conclusion that ABP 959 and eculizumab RP exhibit highly similar inhibition of C5 function regardless of the type of serum used. This work presents a full comparison of the effect of C5 inhibition across five complement functional assays. Using this approach to confirm functional similarity of ABP 959 with eculizumab RP contributes to the TOE for biosimilarity and provides support for extrapolation based on inhibition of C5 function to other rare disease indications approved for eculizumab RP. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s00277-023-05439-4. |
format | Online Article Text |
id | pubmed-10640484 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Springer Berlin Heidelberg |
record_format | MEDLINE/PubMed |
spelling | pubmed-106404842023-11-14 Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD McBride, Helen J. Frazer-Abel, Ashley Thiemann, Sandra Lehto, Sonya G. Hutterer, Katariina M. Liu, Jennifer Ann Hematol Original Article ABP 959 is being developed as a biosimilar to Soliris® (eculizumab) reference product (RP), which was approved under orphan designation for a group of rare diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). Development of biosimilars for therapeutics approved for rare disease indications must provide scientific rationale based on the totality of evidence (TOE). To support the TOE and the scientific justification for extrapolation to all approved indications for eculizumab RP, including but not limited to aHUS and NMOSD, we utilized simulated ex-vivo pharmacodynamic (PD) assessments to compare the complement component 5 (C5) inhibitory activity of ABP 959 and the RP. Hemolysis activity of CH50 and AH50, and Wieslab CP, AP, and LP endpoints represent the three complement activation pathways (classical, alternative, and lectin), all of which share the terminal pathway and require C5 for activity. These endpoints were evaluated in normal serum, simulated aHUS serum, and simulated NMOSD serum to provide a robust comparison. The results support the conclusion that ABP 959 and eculizumab RP exhibit highly similar inhibition of C5 function regardless of the type of serum used. This work presents a full comparison of the effect of C5 inhibition across five complement functional assays. Using this approach to confirm functional similarity of ABP 959 with eculizumab RP contributes to the TOE for biosimilarity and provides support for extrapolation based on inhibition of C5 function to other rare disease indications approved for eculizumab RP. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s00277-023-05439-4. Springer Berlin Heidelberg 2023-10-10 2023 /pmc/articles/PMC10640484/ /pubmed/37817009 http://dx.doi.org/10.1007/s00277-023-05439-4 Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . |
spellingShingle | Original Article McBride, Helen J. Frazer-Abel, Ashley Thiemann, Sandra Lehto, Sonya G. Hutterer, Katariina M. Liu, Jennifer Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD |
title | Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD |
title_full | Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD |
title_fullStr | Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD |
title_full_unstemmed | Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD |
title_short | Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD |
title_sort | functional similarity of abp 959 and eculizumab in simulated serum models of ahus and nmosd |
topic | Original Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10640484/ https://www.ncbi.nlm.nih.gov/pubmed/37817009 http://dx.doi.org/10.1007/s00277-023-05439-4 |
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