Europe Recommends Stem Cell Therapy for Blood Cancers
At its June 2025 meeting, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) gave a recommendation for conditional marketing authorization in the European Union for Zemcelpro (dorocubicel/allogeneic umbilical cord-derived CD34- cells non-expanded, Cordex Biologics International Limited) to treat adults with hematologic malignancies.
A conditional marketing authorization is granted to a medicinal product that fulfils an unmet medical need when the benefit to public health of immediate availability outweighs the risk inherent in the fact that additional data are still required.
Hematologic malignancies include leukemias, lymphomas, myelodysplastic syndrome, and myelomas. The only potential curative treatment option for several of these cancers is allogeneic hematopoietic stem cell transplantation (allo-HSCT). This type of transplant involves using donated stem cells to replace the recipient's bone marrow cells to form new bone marrow that produces healthy blood cells.
Zemcelpro can be used in patients requiring an allo-HSCT following myeloablative conditioning — chemotherapy and/or radiotherapy — for whom no other type of suitable donor cells is available, the agency said.
Novel Cell Therapy
Zemcelpro is a novel cell therapy containing expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit.
By increasing the number of cells, Zemcelpro makes the stem cells from a small cord blood unit more effective.
The benefit of Zemcelpro is its ability to induce neutrophil and platelet engraftment, as observed in two single-arm, open-label, phase 2 clinical studies.
The decision by the CHMP was largely based on a pooled analysis of these studies, which included 25 patients. In total, 84% of patients achieved neutrophil engraftment within a median time of 20 days, and 68% of patients achieved platelet engraftment within a median time of 40 days.
In its overall assessment of the available data, the Committee for Advanced Therapies (CAT), EMA's expert committee for cell- and gene-based medicines, found that the benefits of Zemcelpro outweighed the possible risks in patients with hematologic malignancies requiring allo-HSCT for whom no matched donor cells were available.
Further Study Results Requested
Zemcelpro will be available as a ≥ 0.23 x 106 viable CD34+ cells/mL / ≥ 0.53 x 106 viable CD3+ cells/mL dispersion for infusion.
The most common side effects with the treatment include lymphopenia, infections, anemia, neutropenia, thrombocytopenia, leukopenia, hypogammaglobulinemia, febrile neutropenia, hypertension, engraftment syndrome, pneumonia, and graft-vs-host disease (GvHD).
Zemcelpro was supported through EMA's Priority Medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs.
To confirm the safety and efficacy of the treatment, the company has been requested to submit long-term follow-up results of the single-arm studies, and conduct a randomized controlled study as well as a study based on a patient registry.
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