Finding a treatment for hematological malignancies can be a challenging as it mainly depends on finding a suitable donor & if they are lucky enough to find a donor through a procedure called allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Zemcelpro was developed as an advanced cell therapy. It contains stem cells derived from a donor’s cord blood, including a portion that has been expanded in the lab (dorocubicel) to increase the total number of cells. This process enhances the effectiveness of smaller cord blood units, making them a more viable option for patients without a suitable donor match.

Zemcelpro received support through the EMA’s PRIority MEdicines (PRIME) scheme, which offers early guidance to therapies that address critical unmet medical needs. The positive recommendation was based on results from two open-label studies involving 25 patients. Among them, 84% achieved neutrophil engraftment within a median of 20 days, and 68% achieved platelet engraftment within a median of 40 days demonstrating promising potential for faster and more effective transplant recovery.

In a larger group of 116 patients, common side effects of Zemcelpro included low blood cell and antibody levels, high blood pressure, infections, and engraftment syndrome. Acute GvHD occurred in 60% of patients, while chronic GvHD was seen in 13%. These are addressed through outlined safety measures.

The EMA’s CAT and CHMP concluded that Zemcelpro’s benefits outweigh its risks for patients with blood cancers needing allo-HSCT without a matched donor. It received conditional marketing authorisation to allow earlier access, with the company required to provide long-term data, a controlled trial, and a registry study.

Learn more: New stem cell therapy to treat patients with blood cancers | European Medicines Agency (EMA)