UM171 Cell Therapy Earns Positive CHMP Opinion for Hematological Malignancies

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The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending the approval of UM171 cell therapy (Zemcelpro) for the treatment of adult patients with hematological malignancies who require allogeneic hematopoietic stem cell transplantation after myeloablative conditioning and who do not have other suitable donor cells available.1

Notably, the European Commission is expected to announce its final decision within approximately 2 months, which will apply to all 27 European Union (EU) Member States, Iceland, Norway, and Liechtenstein.

If approved by the European Commission, UM171 cell therapy will be the first and only therapy in the EU with marketing authorization for this patient population. The therapy is a novel cryopreserved hematopoietic stem cell transplantation product that comprises 2 components: UM171-expanded CD34-positive cells (dorocubicel) and unexpanded CD34-negative cells, both derived from the same cord blood unit. Of note, the positive CHMP opinion was supported by the conditional marketing authorization application for UM171 cell therapy, and additional filings are planned with other regulatory agencies in the United States, Canada, the United Kingdom, and Switzerland.

“With today’s positive opinion, we are closer to bringing the life-changing potential of [UM171 cell therapy] to patients with at-risk hematological malignancies in the EU,” David Millette, chief executive officer of ExCellThera, the developer of UM171 cell therapy, stated in a news release. “We are proud to bring our transformative cell therapy innovation to patients who continue to have unmet medical needs.”

UM171 cell therapy has been evaluated in 2 independent prospective phase 2 trials (NCT02668315) for the treatment of patients with high-risk hematological malignancies, including acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), and myelodysplastic syndrome (MDS).2 Patients included on the studies were required to have adequate organ function and a comorbidity index defined by the transplant center. UM171 cell therapy was administered in a 7-day expanded and cryopreserved cord blood after either a myeloablative or a mid-intensity conditioning regimen; all patients received tacrolimus and mycophenolate mofetil for graft-vs-host-disease (GVHD) prophylaxis.

Among the 52 patients enrolled on both studies, 2 patients did not receive UM171 and were removed from the final analysis. At a median follow-up of 24 months (range, 0.7-39), the probability of 2-year overall survival (OS) and progression-free survival (PFS) was 67% (95% CI, 54%-82%) and 63% (95% CI, 50%-79%), respectively. Additionally, the probability of 2-year OS for patients with second or third transplants and TP53 mutation was 64% (95% CI, 43%-96%) and 40% (95% CI, 19%-86%), and the 2-year probability of PFS was 51% (95% CI, 29%-87%) and 40% (95% CI, 19%-86%), respectively.

Furthermore, the cumulative incidence of nonrelapse mortality at 2 years was 19% (95% CI, 8%-31%), and the incidence of relapse was 18% (95% CI, 6%-29%), respectively. Regarding safety, the incidence of grade 2 to 4 and grade 3/4 acute GVHD was 69% (95% CI, 56%-82%) and 16% (95% CI, 6%-27%), respectively. The rate of moderate to severe chronic GVHD was 7% (95% CI, 1%-15%), which led to a chronic relapse-free survival rate of 55% (95% CI, 42%-72%).

“Each year, thousands of people in Europe are diagnosed with hematological malignancies requiring an allogeneic hematopoietic stem cell transplantation, and it’s an upsetting reality that a number of them don’t have access to suitable donor-derived stem cells,” Guy Sauvageau, MD, PhD, FRCP(C), FRSC, chief security officer and founder of ExCellThera, noted in the news release.1

References

1. Zemcelpro (UM171 cell therapy) receives positive CHMP opinion for treatment of blood cancer patients without access to suitable donor cells. News release. ExCellThera. June 19, 2025. Accessed June 20, 2025. https://excellthera.com/news/zemcelpro-um171-cell-therapy-receives-positive-chmp-opinion-for-treatment-of-blood-cancer-patients-without-access-to-suitable-donor-cells/
2. Milano F, Sauvageau G, Thauvette G, et al. Infusion of UM171-expanded cord blood led to excellent survival in patients with high-risk leukemias: results from two independent phase II studies. Blood. 2023;142(suppl 1):1040. doi:10.1182/blood-2023-187446