European Commission Approves Zemcelpro®: First Sole Cell Therapy for Blood Cancer Patients Lacking Suitable Donors
Zemcelpro® offers a new stem cell transplant option for adults with blood cancers unable to find compatible donors in Europe.
ExCellThera and its subsidiary Cordex Biologics have received conditional marketing authorization from the European Commission for Zemcelpro®, a cell therapy indicated for adults with haematological malignancies who require allogeneic haematopoietic stem cell transplantation following myeloablative conditioning, but lack access to suitable donor cells. This authorization covers all European Union member states, as well as Iceland, Norway, and Liechtenstein.
Zemcelpro® (also known as UM171 Cell Therapy) is a cryopreserved haematopoietic stem cell transplant product that contains two components from the same cord blood unit: UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells. The therapy is designed to expand access to allogeneic stem cell transplantation for patients with life-threatening blood cancers such as leukemias and myelodysplastic syndromes, particularly for those unable to find a compatible donor in time. Each year, over 10,000 patients in Europe are diagnosed with these conditions and may require a transplant, yet some are unable to proceed due to donor unavailability or timing constraints. This is a particular challenge for individuals from ethnic minority backgrounds, who are disproportionately affected by donor shortages.
Key Points:
- Zemcelpro® is intended for adults with haematological malignancies needing allogeneic stem cell transplantation after myeloablative conditioning, when no suitable donor cells are available.
- The product is a one-time, personalized cell therapy developed from a single cord blood unit.
- Conditional authorization enables access in the EU, Iceland, Norway, and Liechtenstein, with actual availability subject to national reimbursement procedures.
- Cordex Biologics is engaging with health authorities and transplant centers to establish early access programs and future treatment networks.
Dr. Fabio Ciceri, Professor of Hematology at Vita-Salute San Raffaele University in Milan, Italy, commented: “The curative potential of allogeneic stem cell transplantation is still limited by the access to a suitable donor. The access to stem cell donation according to the stringent disease course is one of the major limiting factors for success. UM171 Cell Therapy offers an option timely available for patients in need.”
Dr. J. (Jurjen) Versluis, Internist-Hematologist and Principal Investigator at Erasmus MC, Rotterdam, The Netherlands, stated: “Each year, thousands of people across Europe are diagnosed with blood cancers that require potentially life-saving allogeneic stem cell transplantation. Yet some are left without access to suitable donor-derived blood stem cells, facing a critical and unmet medical need.”
David Millette, CEO of ExCellThera and Cordex Biologics, said: “This authorization marks a pivotal milestone in delivering on the therapeutic promise of Zemcelpro® for adults battling life-threatening haematological malignancies. As the first and only cell therapy approved for patients without access to suitable donor cells, Zemcelpro® offers a vital new stem cell transplant option — and renewed hope — for those who have been left without one.”
Cordex Biologics is actively seeking partnerships to accelerate commercialization of Zemcelpro® in Europe and other markets. Additional regulatory submissions are planned for the US, Canada, the UK, and Switzerland. In the interim, the company is working with national health authorities to explore early access for eligible patients and is building a network of treatment centers.
Zemcelpro® has received orphan drug designation and regenerative medicine advanced therapy (RMAT) status from the US FDA, as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification, and PRIME designation from the European Medicines Agency (EMA). The product has been evaluated in 120 patients in clinical trials across the US, Europe, and Canada, with a pivotal Phase 3 trial planned for patients with high-risk and very high-risk leukemias and myelodysplastic syndromes. Studies are also underway to assess use in pediatric and non-malignant patient populations.
The safety and efficacy of Zemcelpro® have not yet been established by other regulatory agencies outside the European Union.
Conditional Marketing Authorizations (CMAs) are granted to medicines that address serious or life-threatening diseases with unmet medical need, allowing earlier access while additional clinical data is collected. CMAs are valid for one year and are subject to annual renewal and ongoing regulatory review.
For further information about Zemcelpro®, including the EU Summary of Product Characteristics, visit the ExCellThera website.
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