– A novel cell therapy for life-threatening blood cancers has secured a significant regulatory advancement in Germany, potentially accelerating access for patients with limited treatment options. Zemcelpro® (dorocubicel), developed by ExCellThera Inc. And its subsidiary Cordex Biologics, has been granted Status 1 listing by the NUB (Neue Untersuchungs- und Behandlungsmethoden) program, the companies announced today.
The NUB designation allows German hospitals to immediately apply for temporary, supplementary reimbursement for Zemcelpro®, bridging the gap while broader, long-term reimbursement pathways are established. Notably, 220 hospitals across Germany have already expressed interest in accessing the therapy through this mechanism in the current calendar year, signaling substantial clinical demand.
Zemcelpro®, also known as UM171 Cell Therapy, is a personalized, cryopreserved haematopoietic stem cell transplantation product. It’s designed for adult patients with haematological malignancies requiring allogeneic haematopoietic stem cell transplantation (allo-HSCT) – a procedure where patients receive stem cells from a donor – but for whom a suitable donor is unavailable following myeloablative conditioning, a high-dose chemotherapy regimen. The therapy contains two key components: UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, both derived from the same cord blood unit.
“NUB Status 1 represents a significant milestone for ExCellThera and Cordex and reflects both the innovative nature of Zemcelpro® (dorocubicel) and its benefit in addressing a pressing unmet medical need in allogeneic haematopoietic stem cell transplantation,” said David Millette, CEO of ExCellThera, in a company statement. The company emphasizes that Zemcelpro® is indicated for patients with life-threatening blood cancers who have limited therapeutic options.
The regulatory win in Germany follows recent conditional marketing authorization from the European Commission for the treatment of these challenging cases. Cordex plans to work closely with leading German stem cell transplant centers to support individual NUB applications and expand clinical adoption. The company will also invest in post-market registry participation and generate further evidence and economic data to support longer-term reimbursement and integration into clinical guidelines.
The availability of Zemcelpro® in other European countries will depend on the completion of national reimbursement procedures. Cordex is proactively engaging with national health authorities to enable early access for eligible patients and is working to establish a network of treatment sites across Europe.
Beyond Europe, ExCellThera and Cordex are planning regulatory filings with health authorities in the US, Canada, the UK, and Switzerland. The companies are also actively seeking strategic partnerships to accelerate the commercialization of Zemcelpro® in international markets.
Zemcelpro® has been evaluated in 120 patients with haematologic malignancies in clinical trials across the United States, Europe, and Canada. The therapy 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.
Clinical trials have focused on patients with high and very high-risk acute leukemias and myelodysplasias, often with limited treatment options and poor survival rates, including those with TP53 mutations or requiring a second transplant. A pivotal Phase 3 trial in this patient population is planned.
ExCellThera’s proprietary EnhanceTM platform, which underpins Zemcelpro®, is designed to deliver a greater dose of functional therapeutic stem cells by expanding haematopoietic stem cells (HSCs) from any source and mitigating the negative effects of cell culture or gene editing. The platform utilizes the proprietary molecule UM171, which has a first-in-class mechanism of action for ex vivo expansion and metabolic fitness of HSCs.
The company is also investigating the use of Zemcelpro® in other patient populations, including pediatric patients and those with non-malignant haematological diseases.
