Motixafortide: First Approval
Funding The preparation of this review was not supported by any external funding.
Authorship and Conflict of interest During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Sheridan M. Hoy is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.
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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.
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