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Jasper Therapeutics Announces European Union Orphan Drug Designation for Briquilimab as a Conditioning Treatment for Patients Prior to Receiving a Stem Cell Transplant
REDWOOD CITY, Calif., Jan. 04, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on transforming the field of
About this update from Jasper Therapeutics, Inc.
[{"type":"text","content":"REDWOOD CITY, Calif., Jan. 04, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on transforming the field of hematopoietic cell transplant (HCT) therapies, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted orphan drug designation to briquilimab (formerly known as JSP191), a monoclonal antibody targeting the CD117 (stem cell factor) receptor, for conditioning treatment prior to HCT. Previously, the U.S. Food and Drug Administration granted orphan drug designation to briquilimab in HCT, as well as rare pediatric disease designation for the treatment of severe combined immunodeficiency (SCID). “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. With the orphan designation, the EMA has demonstrated support of briquilimab in Europe and Jasper is committed to advancing this therapy globally.” Jasper is currently conducting clinical studies of briquilimab as a conditioning agent prior to hematopoietic stem cell transplant in patients with SCID, a rare, life-threatening, pediatric disorder affecting an estimated 1/58,000 births in the general population, and separately in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). In these diseases, prognosis is poor for patients and transplant rates are low due to the highly toxic conditioning required. Jasper’s ongoing clinical trial in SCID is evaluating briquilimab as a conditioning agent to enable stem cell transplantation in patients who are either transplant-naive or who received a prior stem cell transplant with a poor outcome. Jasper presented data from this study at multiple scientific conferences, which demonstrated that briquilimab h...