Motixafortide: First Approval

Drugs. 2023 Nov;83(17):1635-1643. doi: 10.1007/s40265-023-01962-w.

Abstract

Motixafortide (APHEXDATM) is a selective C-X-C motif chemokine receptor 4 (CXCR4) inhibitor being developed by BioLineRx under licence from Biokine Therapeutics for the mobilization of haematopoietic stem cells (HSCs) and the treatment of various cancers. On 11 September 2023, motixafortide was approved in the USA for use in combination with filgrastim [granulocyte colony stimulating factor (G-CSF)] to mobilize HSCs to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. Motixafortide has been granted Orphan Drug Designation for the treatment of pancreatic cancer in the EU and the USA, and for the treatment of acute myeloid leukaemia in the USA. Clinical development is ongoing for the mobilization of CD34+ HSCs for gene therapy in patients with sickle cell disease. This article summarizes the milestones in the development of motixafortide leading to this first approval.

Publication types

  • Review

MeSH terms

  • Filgrastim / therapeutic use
  • Granulocyte Colony-Stimulating Factor / pharmacology
  • Granulocyte Colony-Stimulating Factor / therapeutic use
  • Hematopoietic Stem Cell Mobilization
  • Hematopoietic Stem Cell Transplantation*
  • Hematopoietic Stem Cells
  • Humans
  • Immunologic Factors / therapeutic use
  • Multiple Myeloma* / drug therapy
  • Transplantation, Autologous

Substances

  • Granulocyte Colony-Stimulating Factor
  • Filgrastim
  • Immunologic Factors