Stem cell therapeutics (ExCellThera)

ExCellThera’s Zemcelpro gains conditional EC marketing authorisation for blood cancer. Credit: Motortion Films/Shutterstock.com. ExCellThera has announced that the European Commission (EC) has granted conditional marketing authorisation for its Zemcelpro cell therapy to treat blood cancer patients without access to suitable donor cells. The approval was granted to the company’s subsidiary, Cordex Biologics. Zemcelpro is intended for adults with haematological malignancies who require allogeneic haematopoietic stem cell transplantation after myeloablative conditioning. This authorisation enables the distribution of Zemcelpro across European Union (EU) member states, as well as in Iceland, Norway and Liechtenstein. Also referred to as UM171 Cell Therapy, Zemcelpro combines UM171-expanded CD34+ cells and unexpanded CD34- cells derived from the same cord blood unit. It aims to offer a transplant option for patients lacking suitable donor cells. Zemcelpro is a therapy developed to increase access to life-saving transplants, potentially reducing healthcare burdens associated with blood cancers. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence ExCellThera and Cordex Biologics CEO David Millette stated: “This authorisation 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.” The approval follows a positive recommendation from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) in June 2025. The committee acknowledged Zemcelpro’s ability to induce neutrophil and platelet engraftment based on two Phase II clinical studies. Cordex Biologics has tested Zemcelpro on 120 patients in trials across the US, Europe and Canada. The treatment has received several regulatory designations, including orphan drug status from the US Food and Drug Administration (FDA) and PRIority MEdicines (PRIME) designation from the EMA. ExCellThera plans further regulatory submissions in the US, the UK, Canada and Switzerland. Cordex Biologics is engaging with national health authorities to expedite early patient access before reimbursement processes are finalised. The future availability of Zemcelpro will depend on national reimbursement outcomes. A Phase III trial targeting high-risk acute leukaemias and myelodysplasias is planned. The safety profile of Zemcelpro aligns with that of conventional allogeneic stem cell transplantation methods. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic You have a right to withdraw your consent at any time, by clicking here . We may still continue to send you service-related and other non-promotional communications. For more information relating to our privacy practices, we invite you to review our privacy policy . Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

Cordex Biologics' stem cell treatment uses unique technology to multiply the number of cells that can be used from a donor's umbilical cord blood unit. A new treatment option for certain patients with blood cancers has passed muster with the European Commission (EC).Zemcelpro, made by ExCellThera subsidiary Cordex Biologics, is a one-time cell therapy meant to offer a curative treatment for those with hematological malignancies (also known as blood cancers) who don’t have access to necessary donor cells.After winning an endorsement from the Europe’s Committee for Medicinal Products for Human Use (CHMP) in June, the EC has now followed suit with a nod authorizing the therapy to treat adults with blood cancers who require an allogeneic hematopoietic stem cell transplantation following myeloablative conditioning (chemotherapy or radiotherapy) and for whom no other type of suitable donor cells are available.“This authorization marks a pivotal milestone in delivering on the therapeutic promise of Zemcelpro for adults battling life-threatening haematological malignancies,” ExCellThera and Cordex’s CEO David Millette said in an Aug. 27 press release. “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.”Zemcelpro is a unique personalized treatment made up of stem cells derived from a donor’s umbilical cord blood, some of which have been grown and multiplied. Under this approach, Zemcelpro renders the stem cells from a small blood cord unit more effective, according to a June European Medicines Agency release on the CHMP decision. In clinical studies, 21 of the 25 patients that ExCellThera evaluated in a pooled analysis of two single-arm, open-label studies achieved neutrophil engraftment, the term for when donor stem cells successfully produce the white blood cells neutrophils, within a median time of 20 days. The European approval is conditional, meaning the company will have to provide more comprehensive clinical data in the future.A phase 3 study will be initiated “as soon as possible,” according to ExCellThera. Zemcelpro’s potential use in other patient populations, such as pediatric patients and those with non-malignant hematological diseases, is also being investigated.  With the nod, Zemcelpro offers a first-of-its-kind option to many of the thousands of patients who are diagnosed each year in Europe with blood cancers—such as leukemias and myelodysplastic syndromes—as well as the only potential curative option for some blood cancers. Many patients face barriers that prevent them from accessing suitable donor cells, such as a lack of compatible donors or trouble securing cells within the often-short window required for transplant. No official statistics exist to track the number of patients who miss out on potentially curative transplants, but the group includes a disproportionately high number of people from ethnic minority backgrounds, ExCellThera pointed out in its release.Patients who do not receive needed stem cell transplants face disease progression or complications and may need hospitalization and supportive care. Zemcelpro, for its part, "has the potential not only to save lives but also to reduce the healthcare and societal burden associated with these devastating conditions,” the company noted. The drug’s launch hinges on several factors, including wrapping up national reimbursement procedures. Meanwhile, ExCellThera’s Cordex is currently working “closely” with authorities in individual countries to enable early access for eligible patients as the reimbursement talks progress.The company is also in discussions with leading stem cell transplant centers to set up a network of treatment centers for the future administration of Zemcelpro.Beyond Europe, additional regulatory filings are planned for the U.S., Canada, the U.K. and Switzerland, according to the company. Cordex is “actively seeking” strategic partnerships to ramp up the commercialization efforts in Europe and other markets, its parent company said.