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The agency recommends Zalmoxis, a new cell-based therapy to support stem cell transplantation in patients with high-risk blood cancer.
On June 24, 2016, the European Medicines Agency (EMA) announced its Committee for Medicinal Products for Human Use (CHMP) was recommending orphan drug Zalmoxis for conditional marketing authorization in the European Union as a new advanced therapy medicinal product (ATMP). Zalmoxis is an add-on treatment for “adult patients receiving a haploidentical hematopoietic stem cell transplant (HSCT) for various types of blood cancer to aid immune reconstitution and reduce the risk of graft-versus-host disease,” according to EMA. Zalmoxis was give orphan medicinal product designation in 2003.
Conditional marketing authorization, which allows marketing authorization before the availability of confirmatory clinical trial data, is given when the agency determines that the benefits of a therapy outweigh the risks associated with a lack of comprehensive data. The applicant for Zalmoxis must provide results from a comparable Phase III trial as part of the conditional authorization. EMA’s CHMP and Committee on Advanced Therapies (CAT) will review annually the benefits and risks of the drug to determine continued conditional marketing authorization.
EMA states that, with a haploidentical HSCT, the patient receives hematopoietic stem cells from a partially matched donor to help the bone marrow produce healthy blood cells. Haploidentical HSCT is used in the treatment of hematological malignancies, such as leukemia and lymphoma. Transplants from partially-matched donors, which are more readily available than perfect matches, carry a higher risk of transplanted cells attacking the recipient’s organs.
The T cells in Zalmoxis, which are taken from the stem cell donor, have been separated from the rest of the cells in the transplant and have been genetically modified to include a ‘suicide gene’ called HSV-TK. This suicide gene makes the T cells susceptible to the drug ganciclovir, which will kill the T cells that have the suicide gene if the patient develops graft-versus-host disease.
Zalmoxis was given to 30 patients with blood cancers who had haploidentical HSCT in a trial. Twenty-three of these patients had their immune systems restored. Ten patients developed graft-versus-host disease, and nine of those were given ganciclovir. None of the patients died or suffered long-term serious effects from graft-versus-host disease. “When overall survival rates from a total of 45 patients (30 from this trial and 15 from a second ongoing trial) treated with Zalmoxis were compared to rates from databases of patients who have undergone haploidentical HSCT, survival rates were higher for patients who received Zalmoxis (49% survival after one year, compared to 37% for patients who did not receive Zalmoxis),” EMA stated in a press release.
The CHMP recommendation will be sent the the European Commission for EU-wide market authorization.