HSCT | Briquilimab Granted Orphan Drug Designation By The European Commission

Jasper Therapeutics announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted orphan drug designation to briquilimab, a monoclonal antibody targeting the CD117 (stem cell factor) receptor, for conditioning treatment prior to haematopoietic stem cell transplant (HSCT).

Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to briquilimab in HCT, as well as rare pediatric disease designation for the treatment of severe combined immunodeficiency (SCID).

Only a day earlier to the EMA’s orphan designation to briquilimab, Jasper Therapeutics had reported positive clinical trial results from the first three subjects in a Phase I/II clinical trial of briquilimab as a conditioning therapy in sickle cell disease (SCD) and β-thalassaemia patients.

The investigator-initiated trial has been designed to assess the addition of the antibody to an existing bone marrow transplantation regimen in these individuals, considered at high risk for complications from, or are not eligible for, standard myeloablative hematopoietic stem cell transplants. The addition of briquilimab is being investigated as a potential way to achieve a higher, healthy donor stem cell engraftment percentage, without elevated toxicity.

At present, the trial is actively enrolling participants at National Heart, Lung, and Blood Institute (NHLBI). The trial’s primary objective is to determine if the addition of briquilimab would increase the proportion of patients with donor myeloid chimerism ≥98% after transplant at one-year. All three sickle cell study participants who received treatment with briquilimab successfully engrafted and no severe adverse events related to the treatment were observed.

“The EMA’s decision to grant orphan drug designation to briquilimab highlights the clear need for non-genotoxic, targeted conditioning for patients receiving hematopoietic stem cell transplant,” said Ronald Martell, President and Chief Executive Officer of Jasper Therapeutics. “Transplants have the potential to cure several hematologic cancers and genetically inherited diseases. However, the toxicities associated with genotoxic conditioning needed to prepare patients for these procedures often limit their use. We believe that briquilimab has the potential to fill this gap, effectively expanding access to curative stem cell transplant across a range of indications.” 

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