ExCellThera Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending conditional marketing authorization for Zemcelpro for treating adults with haematological malignancies requiring allogeneic haematopoietic stem cell transplantation following myeloablative conditioning when no other suitable donor cells are available. The European Commission is expected to make a final decision within approximately two months, with approval applying to all 27 EU Member States, Iceland, Norway and Liechtenstein.
Novel Cell Therapy Technology
Zemcelpro, also known as UM171 Cell Therapy, represents a novel cryopreserved haematopoietic stem cell transplantation product containing two components: UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, both derived from the same cord blood unit. The therapy was developed by Cordex Biologics, a wholly owned subsidiary of ExCellThera.
If approved, Zemcelpro is expected to be the first and only therapy in the EU with marketing authorization for this specific patient population. The therapy would increase access to donor-derived stem cell transplantation, which offers a potentially curative option for haematologic malignancies, including leukemias and myelodysplastic syndromes.
Addressing Significant Unmet Medical Need
Every year in Europe, over 10,000 new cases of patients with haematological malignancies require bone marrow transplant, and a number of these patients do not have access to suitable donor cells for various reasons, including the absence or unavailability of suitably matched donors.
"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.
Clinical Development and Regulatory Status
Zemcelpro has been evaluated in 120 patients with haematologic malignancies in clinical trials across the United States, Europe and Canada. The therapy has received multiple regulatory designations, including 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.
The therapy 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 current standard of care. This includes 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.
Safety Profile and Future Plans
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. The use of Zemcelpro in other patient populations, including pediatric patients and patients with non-malignant haematological diseases, is also being explored.
"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."
Additional filings are planned for Zemcelpro with other health authorities, including in the US, Canada, the UK, and Switzerland. The product is not yet approved for marketing by the EMA and remains subject to European Commission decision, with safety and efficacy not yet established by other regulatory agencies such as the FDA and Health Canada.
