The European Commission has granted conditional marketing authorization to ExCellThera’s stem cell therapy for adults with hematological malignancies requiring an allogeneic hematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available.
A number of blood cancer patients that require stem cell transplants are unable to access suitable donor cells due to lack of a compatible donor or the inability to secure donor cells within the therapeutic window for transplant. Zemcelpro, developed by ExCellThera subsidiary Cordex Biologics, is designed to be a one-time cell therapy with curative intent.
Zemcelpro is a novel personalized cryopreserved hematopoietic stem cell transplantation product containing two components: UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit.
Inphase 2 trials, the therapy demonstrated excellent clinical outcomes at two years for a high and very high-risk cohort of patients with leukemias and myelodysplasias, including strong rates of overall survival (67%) and progression-free survival (63%), as well as low rates of non-relapse mortality (19%), relapse (18%) and moderate-to-severe chronic GVHD (7%).
The therapy has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designations from the FDA as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the EMA. Additional regulatory filings are planned with other health authorities, including in the U.S., Canada, the UK, and Switzerland.
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