Introducing the Biosimilar Paradigm to Neurology: The Totality of Evidence for the First Biosimilar Natalizumab

BioDrugs. 2024 Nov;38(6):755-767. doi: 10.1007/s40259-024-00671-4. Epub 2024 Sep 30.

Abstract

A biosimilar medicine is a successor to a reference ('originator'/'original-brand') biologic medicine brought to market once the patent and exclusive marketing rights for the reference have expired. Biosimilar natalizumab (PB006 [biosim-NTZ]; developed by Polpharma Biologics S.A. and marketed globally as Tyruko®; Sandoz) has been developed as a successor to reference natalizumab (Tysabri® [ref-NTZ]; Biogen) and is the first US Food and Drug Administration (FDA)-approved and European Medicines Agency (EMA)-approved biosimilar in neurology. As per the FDA and EMA indications for ref-NTZ, biosim-NTZ is approved to treat relapsing forms of multiple sclerosis (USA, EU) and Crohn's disease (USA only). Approval of biosim-NTZ was based on the 'totality of evidence', a comprehensive body of data collected during the development process, demonstrating similarity to its reference medicine. The foundational step of demonstrating structural and functional similarity between biosim-NTZ and ref-NTZ confirmed identical primary and indistinguishable higher order structures, as well as matching binding affinity to α4β1/α4β7 integrins. Following the confirmation of matching structure and function, pharmacokinetic/pharmacodynamic similarity of biosim-NTZ to ref-NTZ in healthy subjects was demonstrated, with no clinically meaningful differences identified in safety and immunogenicity. A comparative, double-blind, randomized study (Antelope) was also conducted in patients with relapsing-remitting multiple sclerosis and demonstrated matching efficacy, safety, and immunogenicity with no clinically meaningful differences between biosim-NTZ and ref-NTZ. This review presents the totality of evidence that confirmed the biosimilarity of biosimilar natalizumab to its reference medicine, which supported its approval by the FDA and the EMA. [Graphical plain language summary available].

Publication types

  • Review

MeSH terms

  • Biosimilar Pharmaceuticals* / pharmacokinetics
  • Biosimilar Pharmaceuticals* / pharmacology
  • Biosimilar Pharmaceuticals* / therapeutic use
  • Crohn Disease / drug therapy
  • Drug Approval
  • Humans
  • Multiple Sclerosis / drug therapy
  • Multiple Sclerosis, Relapsing-Remitting / drug therapy
  • Natalizumab* / pharmacology
  • Natalizumab* / therapeutic use
  • Neurology
  • United States
  • United States Food and Drug Administration

Substances

  • Natalizumab
  • Biosimilar Pharmaceuticals