EMA Conditionally Authorizes Stem Cell Therapy for Blood Cancers

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The European Medicines Agency (EMA) announced on June 20, 2025 that it has recommended that Zemcelpro (dorocubicel/unexpanded umbilical cord cells) be granted conditional marketing authorization for the treatment of adults with hematological malignancies (blood cell cancers). Zemcelpro is used to treat patients that require allogeneic hematopoietic stem cell transplantation following chemotherapy or radiotherapy when no other type of suitable donor cells is available.

The cells used for this type of therapy are from a matched donor, sometimes a relative of the patient. However, the number of cells is often too low for engraftment. Zemcelpro contains stem cells from a donor’s umbilical cord blood that are grown and multiplied, making the stem cells more effective by increasing the number of cells.

The EMA recommendation was based on analysis of two single arm, open-label studies that included 25 patients. The majority of patients in the studies (84%) achieved successful establishment of the donor cells in the recipient’s bone marrow and produced neutrophils within a median time of 20 days. In addition, platelet engraftment was achieved in 68% of patients within a median time of 40 days.

Side effects of Zemcelpro that were observed in a wider pool of 116 patients included low levels of various types of blood cells and antibodies, high blood pressure, infections, and the inflammatory condition, engraftment syndrome. Acute graft-versus-host disease (GvHD) was reported in 60% of patients, chronic GvHD was reported in 13% of patients. However, EMA’s Committee for Advanced Therapies decided that the benefits outweigh the risks associated with Zemcelpro.

Conditional marketing authorizations provides early access to drugs that treat unmet needs by allowing the agency to recommend a drug for authorization with fewer data than are usually required. To confirm the safety of the drug, the sponsor company, Cordex Biologics International Limited, must submit long-term follow-up study results, conduct a randomized a controlled study, and perform a study based on a patient registry. EMA’s recommendation, which was supported through the PRIority MEdicines scheme, now goes to the European Commission for adoption. Zemcelpro was also designated as an orphan drug in April 2020 for the treatment in hematopoietic stem cell transplantation. EMA’s Committee for Orphan Medicinal Products will evaluate if that designation should continue.

According to EMA, “hematological malignancies are blood cell cancers categorized depending on where they are first detected and include leukemias (blood), lymphomas (lymph nodes), myelodysplastic syndrome and myelomas (bone marrow). They are frequently diagnosed cancers, and the only potential curative treatment option for several of these cancers is 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” (1).

Dorocubicel and non-expanded CD34 cells, the active substances in Zemcelpro, are stem cells from umbilical cord blood. “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. Once infused to the patient, the cells from Zemcelpro migrate to the bone marrow where they divide, mature and differentiate in all hematological cell lineages,” EMA stated (2).

References

1. EMA. New Stem Cell Therapy to Treat Patients with Blood Cancers. Press Release. June 20, 2025.

2. EMA. Zemvelpro. Ema.europa.eu (accessed June 23, 2025). https://www.ema.europa.eu/en/medicines/human/EPAR/zemcelpro