On June 19, 2025 ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, reported the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending granting conditional marketing authorization for Zemcelpro for the treatment of adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available (Press release, ExCellThera, JUN 19, 2025, View Source [SID1234654009]). The European Commission (EC) is expected to make a final decision within approximately two months following CHMP recommendation, and the decision will apply to all 27 European Union (EU) Member States, Iceland, Norway and Liechtenstein.
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Zemcelpro, also known as UM171 Cell Therapy, is a novel cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit.
If approved, Zemcelpro is expected to be the first and only therapy in the EU with marketing authorization for adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. Every year in Europe there are over 10,000 new cases of patients with haematological malignancies, including leukemias and myelodysplastic syndromes, requiring bone marrow transplant, and a number of these patients do not have access to suitable donor cells for different reasons, including the absence or unavailability of suitably matched donors.
The positive CHMP opinion was based on the conditional Marketing Authorization Application (MAA) for Zemcelpro. Additional filings are planned for Zemcelpro with other health authorities, including in the US, Canada, the UK, and Switzerland.
"Each year, thousands of people in Europe are diagnosed with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation, and it’s an upsetting reality that a number of them don’t have access to suitable donor-derived stem cells," said Dr. Guy Sauvageau, CSO and Founder of ExCellThera.
"With today’s positive opinion, we are closer to bringing the life-changing potential of Zemcelpro to patients with at-risk haematological malignancies in the EU," said David Millette, CEO of ExCellThera. "We are proud to bring our transformative cell therapy innovation to patients who continue to have unmet medical needs."
The safety of Zemcelpro is consistent with the well-characterized safety profile of conventional allogeneic blood stem cell transplantation for haematological malignancies following myeloablative conditioning.
ExCellThera extends its sincere gratitude to the patients and investigators who have contributed to the clinical development of Zemcelpro.
About Zemcelpro
Zemcelpro, also known as UM171 Cell Therapy, is a novel cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit.
Zemcelpro, developed by Cordex Biologics, a wholly owned subsidiary of ExCellThera, has been evaluated in 120 patients with haematologic malignancies in clinical trials in the United States, Europe and Canada. Zemcelpro 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.
Zemcelpro has been tested in Phase 2 trials in patients with high and very high-risk acute leukemias and myelodysplasias who have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care, including patients with patients with TP53 mutations or other genetic abnormalities, patients requiring a second transplant, and patients with refractory or active disease. A pivotal Phase 3 trial in this patient population will be initiated as soon as possible.
The use of Zemcelpro in other patient populations, including pediatric patients and patients with non-malignant haematological diseases, is also being explored.
The product is not yet approved for marketing by the EMA and remains subject to European Commission decision. Its safety and efficacy have not been established by other regulatory agencies, such as the FDA and Health Canada.