Rother RP, Rollins SA, Mojcik CF, Brodsky RA, Bell L. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol. 2007;25(11):1256–64. https://doi.org/10.1038/nbt1344.

Article  CAS  PubMed  Google Scholar 

US Food and Drug Administration. Approval package for application number: 125166s431. 2019. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/125166Orig1s431.pdf. Accessed 8 Aug 2024.

European Medicines Agency. Soliris (eculizumab): an overview of Soliris and why it is authorised in the EU 2009. 2023. Available from: https://www.ema.europa.eu/en/documents/overview/soliris-epar-medicine-overview_en.pdf. Accessed 8 Aug 2024.

Wijnsma KL, Ter Heine R, Moes D, Langemeijer S, Schols SEM, Volokhina EB, et al. Pharmacology, pharmacokinetics and pharmacodynamics of eculizumab, and possibilities for an individualized approach to eculizumab. Clin Pharmacokinet. 2019;58(7):859–74. https://doi.org/10.1007/s40262-019-00742-8.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Al-Ani F, Chin-Yee I, Lazo-Langner A. Eculizumab in the management of paroxysmal nocturnal hemoglobinuria: patient selection and special considerations. Ther Clin Risk Manag. 2016;12:1161–70. https://doi.org/10.2147/TCRM.S96720.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Ferreira VP, Pangburn MK. Factor H–mediated cell surface protection from complement is critical for the survival of PNH erythrocytes. Blood. 2007;110(6):2190–2. https://doi.org/10.1182/blood-2007-04-083170.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Griffin M, Kelly R, Pike A. A review of the treatment landscape in paroxysmal nocturnal haemoglobinuria: where are we now and where are we going? Ther Adv Rare Dis. 2020;1:2633004020959349.

Article  PubMed  PubMed Central  Google Scholar 

Socie G, Caby-Tosi MP, Marantz JL, Cole A, Bedrosian CL, Gasteyger C, et al. Eculizumab in paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome: 10-year pharmacovigilance analysis. Br J Haematol. 2019;185(2):297–310. https://doi.org/10.1111/bjh.15790.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011;117(25):6786–92. https://doi.org/10.1182/blood-2011-02-333997.

Article  CAS  PubMed  Google Scholar 

Avila Bernabeu AI, Cavero Escribano T, Cao VM. Atypical hemolytic uremic syndrome: new challenges in the complement blockage era. Nephron. 2020;144(11):537–49. https://doi.org/10.1159/000508920.

Article  CAS  PubMed  Google Scholar 

Afshar-Kharghan V. Atypical hemolytic uremic syndrome. Hematology. 2016;2016(1):217–25. https://doi.org/10.1182/asheducation-2016.1.217.

Article  PubMed  PubMed Central  Google Scholar 

Yoshida Y, Kato H, Ikeda Y, Nangaku M. Pathogenesis of atypical hemolytic uremic syndrome. J Atheroscler Thromb. 2019;26(2):99–110. https://doi.org/10.5551/jat.RV17026.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Wijnsma KL, Duineveld C, Wetzels JFM, van de Kar N. Eculizumab in atypical hemolytic uremic syndrome: strategies toward restrictive use. Pediatr Nephrol. 2019;34(11):2261–77. https://doi.org/10.1007/s00467-018-4091-3.

Article  PubMed  Google Scholar 

Azoulay E, Knoebl P, Garnacho-Montero J, Rusinova K, Galstian G, Eggimann P, et al. Expert statements on the standard of care in criticallyiIll adult patients with atypical hemolytic uremic syndrome. Chest. 2017;152(2):424–34. https://doi.org/10.1016/j.chest.2017.03.055.

Article  PubMed  Google Scholar 

Legendre CM, Licht C, Muus P, Greenbaum LA, Babu S, Bedrosian C, et al. Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med. 2013;368(23):2169–81. https://doi.org/10.1056/NEJMoa1208981.

Article  CAS  PubMed  Google Scholar 

Raina R, Grewal MK, Radhakrishnan Y, Tatineni V, DeCoy M, Burke LL, et al. Optimal management of atypical hemolytic uremic disease: challenges and solutions. Int J Nephrol Renovasc Dis. 2019;12:183–204. https://doi.org/10.2147/IJNRD.S215370.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Neave L, Gale DP, Cheesman S, Shah R, Scully M. Atypical haemolytic uraemic syndrome in the eculizumab era: presentation, response to treatment and evaluation of an eculizumab withdrawal strategy. Br J Haematol. 2019;186(1):113–24. https://doi.org/10.1111/bjh.15899.

Article  CAS  PubMed  Google Scholar 

Merrill SA, Brittingham ZD, Yuan X, Moliterno AR, Sperati CJ, Brodsky RA. Eculizumab cessation in atypical hemolytic uremic syndrome. Blood. 2017;130(3):368–72. https://doi.org/10.1182/blood-2017-02-770214.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Caetano R, Cordeiro Dias Villela Correa M, Villardi P, Almeida Rodrigues PH, Garcia Serpa Osorio-de-Castro C. Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: the market expansion of eculizumab. PLoS One. 2021;16(3):e0247853. https://doi.org/10.1371/journal.pone.0247853.

IQVIA. The impact of biosimilar competition in Europe 2023. 2023. Available from: https://www.iqvia.com/library/white-papers/the-impact-of-biosimilar-competition-in-europe-2023. Accessed 12 Aug 2024.

Kay J, Schoels MM, Dorner T, Emery P, Kvien TK, Smolen JS, et al. Consensus-based recommendations for the use of biosimilars to treat rheumatological diseases. Ann Rheum Dis. 2018;77(2):165–74. https://doi.org/10.1136/annrheumdis-2017-211937.

Article  CAS  PubMed  Google Scholar 

Milassin A, Fabian A, Molnar T. Switching from infliximab to biosimilar in inflammatory bowel disease: overview of the literature and perspective. Therap Adv Gastroenterol. 2019;12:1756284819842748. https://doi.org/10.1177/1756284819842748.

Article  PubMed  PubMed Central  Google Scholar 

Kvien TK PK, Strand V. The cost savings of biosimilars can help increase patient access and lift the financial burden of health care systems. Semin Arthritis Rheumatol. 2022;52(151939). https://doi.org/10.1016/j.semarthrit.2021.11.009

Moorkens EJEC, Huys I, Declerck P, Simoens S, Vulto AG. Overcoming barriers to the market access of biosimilars in the European Union: the case of biosimilar monoclonal antibodies. Front Pharmacol. 2016;7(193):eCollection2016. https://doi.org/10.3389/fphar.2016.00193.

Article  CAS  Google Scholar 

Barbier LSS, Vulto AG, Huys I. European stakeholder learnings regarding biosimilars. Part II: improving biosimilar use in clinical practice. BioDrugs. 2022;34(6):12. https://doi.org/10.1007/s40259-020-00440-z.

Article  Google Scholar 

Baumgart DC, Misery L, Naeyaert S, Taylor PC. Biological therapies in immune-mediated inflammatory diseases: can biosimilars reduce access inequities? Front Pharmacol. 2019;10:279. https://doi.org/10.3389/fphar.2019.00279.

Article  CAS  PubMed  PubMed Central  Google Scholar 

World Health Organization. Guidelines on evaluation of similar biotherapeutic products (SBPs), Annex 2, TRS No 977. 2010. Available from: https://www.who.int/publications/m/item/sbp-trs-977-Annex-2. Accessed 8 Aug 2024.

US Food and Drug Administration. Biosimilars: overview for health care professionals. 2024. Available from: https://www.fda.gov/drugs/biosimilars/overview-health-care-professionals. Accessed 26 Nov 2024.

European Medicines Agency and European Commission. Biosimilars in the EU: information guide for healthcare professionals. 2019. Available from: https://www.ema.europa.eu/en/documents/leaflet/biosimilars-eu-information-guide-healthcare-professionals_en.pdf. Accessed 8 Aug 2024.

European Medicines Agency. Guideline on similar biological medicinal products. 2014. Available from: https://www.ema.europa.eu/system/files/documents/scientific-guideline/wc500176768_en.pdf. Accessed 8 Aug 2024.

Ryan AM. Frontiers in nonclinical drug development: biosimilars. Vet Pathol. 2015;52(2):419–26. https://doi.org/10.1177/0300985814547282.

Article  CAS  PubMed  Google Scholar 

Wang J, Chow SC. On the regulatory approval pathway of biosimilar products. Pharmaceuticals (Basel). 2012;5(4):353–68. https://doi.org/10.3390/ph5040353.

Article  PubMed  Google Scholar 

US Food and Drug Administration. Considerations in demonstrating interchangeability with a reference product: update. 2024. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-demonstrating-interchangeability-reference-product-update. Accessed 8 Aug 2024.

European Medicines Agency. Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues, revision 1. 2015. Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-similar-biological-medicinal-products-containing-biotechnology-derived-proteins-active-substance-non-clinical-and-clinical-issues-revision-1_en.pdf. Accessed 27 Jun 2024.

US Food and Drug Administration. Biosimilar development, review, and approval. 2022. Available from: https://www.fda.gov/drugs/biosimilars/biosimilar-development-review-and-approval. Accessed 27 Jun 2024.

Gherghescu I, Delgado-Charro MB. The biosimilar landscape: an overview of regulatory approvals by the EMA and FDA. Pharmaceutics. 2020;13(1):48. https://doi.org/10.3390/pharmaceutics13010048.

Article