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Briquilimab granted orphan drug designation by the European Commission

Jan 5, 2023
Learning objective: After reading this article, learners will be able to cite a new development in the treatment of MDS .

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On January 4, 2023, briquilimab, a monoclonal antibody targeting the CD117 receptor, was granted orphan drug designation by the European Commission, as a conditioning treatment prior to hematopoietic stem cell transplantation (HSCT).1 Briquilimab received orphan drug designation from the U.S Food and Drug Administration (FDA) in June 2022.1

As previously reported by the AML Hub, briquilimab is currently being investigated as a conditioning treatment prior to HSCT in patients with acute myeloid leukemia and myelodysplastic syndromes (MDS).1 Briquilimab was found to have a tolerable safety profile, with full donor chimerism in all 24 patients with acute myeloid leukemia or MDS. There were no reports of treatment-related significant adverse events, classical acute Grade II–IV graft-versus-host disease, or transplant mortality within 100 days. Briquilimab is also being assessed as a primary therapy for patients with low-risk MDS and as a conditioning therapy prior to HSCT for patients with severe combined immunodeficiency.1

  1. Jasper Therapeutics. Jasper Therapeutics announces European Union orphan drug designation for briquilimab as a conditioning treatment for patients prior to receiving a stem cell transplant. Published Jan 4, 2023. Accessed Jan 5, 2023.


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