European Medicines Agency Recommends Conditional Marketing Authorisation for Zemcelpro

Strategic Insights -

The European Medicines Agency has recommended granting a conditional marketing authorisation for Zemcelpro, a cell therapy containing stem cells from a donor's umbilical cord blood, to treat adults with haematological malignancies (blood cell cancers) who require an allogeneic haematopoietic stem cell transplantation (allo-HSCT) following myeloablative conditioning. Zemcelpro was supported through EMA's PRIority MEdicines (PRIME) scheme and has shown promising results in treating patients with haematological malignancies, with 84% of patients achieving neutrophil engraftment within a median time of 20 days and 68% achieving platelet engraftment within a median time of 40 days.

Key Takeaways:

  • Zemcelpro is a cell therapy containing stem cells from a donor's umbilical cord blood, which has been grown and multiplied to make the stem cells more effective.
  • The treatment is recommended for adults with haematological malignancies who require an allogeneic haematopoietic stem cell transplantation (allo-HSCT) following myeloablative conditioning.
  • Zemcelpro was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs.
  • The treatment has shown promising results, with 84% of patients achieving neutrophil engraftment within a median time of 20 days and 68% achieving platelet engraftment within a median time of 40 days.
  • The most common side effects observed in a wider pool of 116 patients treated with Zemcelpro include low levels of various types of blood cells and of antibodies that help fight infections, high blood pressure, infections, and engraftment syndrome.
  • The Committee for Advanced Therapies (CAT) and the CHMP found that the benefits of Zemcelpro outweighed the possible risks in patients with haematological malignancies requiring allo-HSCT.
  • The conditional marketing authorisation allows Zemcelpro to be granted marketing authorisation with less complete data than normally expected, if the benefit of the medicine's immediate availability to patients outweighs the risk inherent in the fact that not all the data are yet available.
  • The company has been requested to submit long-term follow-up results of the single arm studies, conduct a randomised controlled study and a study based on a patient registry.

Statistics:

  • 84% of patients achieved neutrophil engraftment within a median time of 20 days.
  • 68% of patients achieved platelet engraftment within a median time of 40 days.
  • 116 patients were treated with Zemcelpro in a wider pool.
  • 60% of patients reported acute graft-versus-host disease (GvHD) up to 100 days after transplantation.
  • 13% of patients reported chronic GvHD appearing up to one year after transplantation.

Sources:

  • European Medicines Agency (EMA)
  • EMA's PRIority MEdicines (PRIME) scheme
  • Committee for Advanced Therapies (CAT)
  • CHMP (EMA's human medicines committee)