Zemcelpro received on 25. August 2025 by the European Commission a conditional approval for the treatment of adult patients with hematological malignancies who require allogeneic hematopoietic stem cell transplantation (allo-HSCT) after myeloablative conditioning and for whom no other type of suitable donor cells is available.
Under the agreement, Avanzanite will lead the marketing in 32 countries, including in the European Economic Area, the United Kingdom and Switzerland. Avanzanite will become the holder of the existing EU approval for Zemcelpro and plans to file applications for authorisation in the UK and Switzerland. ExCellThera retains responsibility for manufacturing and delivery and will continue to lead the global development with a view to a possible extension of indications in certain patients.
In addition, Avanzanite will contribute to the cost of a planned Phase 3 study, in which Zemcelpro is studied with high and very high risk compared to transplants with haploidentic or non-appropriate unrelated donors in adult patients with acute leukemias and myelodysplastic syndromes (MDS). The study is intended to provide confirmatory evidence of efficacy and safety and could support a possible indication expansion.
“This partnership reflects why Avanzanite was founded: to unlock the full potential of innovative drugs for patients with rare diseases in Europe,” said Adam Plich, co-founder and CEO of Avanzanite Bioscience. “We believe that while discovering groundbreaking drugs requires a certain type of genius, making sure these drugs reach the patients who can benefit from it, they do have a different one.
This partnership combines these strengths in a common sense of purpose.” Plich continues, “Our commitment also extends to supporting the funding of the planned Phase 3 trial to gather further evidence and examine the potential of therapy for broader application.”
Zemcelpro has orphan drug status and was part of the European Medicines Agency's PRIority MEdicines (PRIME) programme during development. This program provides promising medicines that address unmet medical needs, early and increased scientific and regulatory support.
“For many years, our team and our cooperation partners have worked tirelessly to advance Zemcelpro from scientific discovery to patient care,” said David Millette, CEO of ExCellThera. “Avanzanite brings a Europe-wide presence and in-depth expertise in the market launch of rare disease medicines and market access. We are convinced that they are the ideal partner to translate the potential of this science into concrete results for patients in need, while continuing to focus on scientific and clinical development.”
“For patients with blood cancer who need a stem cell transplant from a donor, finding suitable donor cells is a crucial step in the course of treatment,” Dr. said. Jurjen Versluis, internist and hematologist at the Erasmus MC Cancer Institute in Rotterdam, Netherlands. “If no other suitable donor cells are available, additional transplant options are important. The Erasmus MC has participated in the clinical trials for the evaluation of Zemcelpro and we are pleased to have contributed to its clinical development.”
The agreement advances Avanzanite’s goal of establishing itself as a European pioneer in the field of rare diseases. With this fifth market launch of a drug for rare diseases within four years, Zemcelpro is expanding the company’s hematological portfolio in addition to the areas of ophthalmology and nephrology with stem cell therapy and hematology oncology. Founded in 2022, Avanzanite now employs more than 110 people across Europe; the company serves more than 400 patients.
About Zemcelpro ®
Zemcelpro is an allogeneic hematopoietic stem cell therapy that is approved in the European Union as a medicine for use in suitable adults who require allogeneic hematopoietic stem cell transplantation (allo-HSCT). It is specially made for each patient from a single cord blood unit and contains two components: expanded CD34+ hematopoietic stem cells using the company’s proprietary UM171 technology (dorocubicel) and non-expanded CD34 cells. After the infusion, the CD34+ stem cells migrate to the bone marrow, where they divide and mature into specialized blood and immune cells. The non-increasing CD34 cells, which consist mainly of CD3+ T cells, play a complementary role by supporting immunoreconstitution and creating a graft-versus-leukemia effect after transplantation.
About allogeneic stem cell transplantation
Each year, around 15,000 adults in the approved areas require allogeneic hematopoietic stem cell transplantation (HSCT) as a potentially curative treatment for blood cancers such as acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndromes (MDS), lymphomas and other diseases. However, a third of these patients urgently need a transplant, but do not have a suitable related donor. Most receive transplants from haploidentic or non-appropriate unrelated donors, which is associated with an increased risk of serious complications, including the graft versus host response (GVHD) and infections that contribute to mortality without relapse. Some may not receive a transplant at all and are at risk of relapse, with their chances of survival being low.
About Avanzanite Bioscience
Avanzanite is a next-generation pharmaceutical company specifically designed to unlock the full potential of rare disease medicines across Europe. Avanzanite was founded in 2022 and is headquartered in Amsterdam, the Netherlands. The company is based on the belief that scientific innovation only has a benefit for patients if the medicines reach those who can benefit from it. Through tailor-made partnerships, biopharmaceutical innovators Avanzanite entrust the European future of their medicines. Avanzanite brings together pan-European expertise, a bold marketing and access strategy, and a powerful implementation to turn Europe's complexity into opportunities, ensure that no patient is left behind, and create sustainable long-term value for partners.
About ExCellThera
ExCellThera is a global leader in improved blood stem cell therapies. Its technology platform is designed to multiply HSCs from any source while counteracting cellular stress associated with cell cultures or gene processing.
For more information, please visit www.excellthera.com
