Athersys has secured a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) for its multipotent adult progenitor cell or MultiStem therapy ‘allogeneic’, for the prevention of graft-versus-host disease (GvHD).
At present, the company is developing its MultiStem cell therapy as a GvHD prophylaxis in patients undergoing allogeneic hematopoietic stem cell (HSC) transplant and is also preparing for its Phase II/III clinical trial.
The US Food and Drug Administration (FDA) has already granted orphan drug status to the MultiStem therapy for the prevention of GvHD.
Athersys head of global regulatory affairs Manal Morsy said: "Together with our US orphan designation for this indication, this EMA designation has the potential to facilitate our development of MultiStem therapy to help patients at risk of GvHD associated with HSC transplantation."
The company said that patients with leukemia or other related malignancies are treated by radiation and chemotherapy, which are administered to destroy cancerous cells, but also substantially impair the blood forming and immune system of the patient.
The procedures are followed by a HSC transplant to reconstitute the immune system in order to combat infection and any remaining malignancy.
According to the company, patients undergoing donor derived, or allogeneic, HSC transplants are at significant risk for serious complications, including GvHD, which results when transplanted immune cells attack several tissues and organs in the patient.
GvHD can be severe and life-threatening and can impact overall treatment requirements and costs.
It is expected that more than 25,000 allogeneic HSC transplants a year are performed in the developed countries.
The company has completed a Phase I clinical trial of the administration of MultiStem cells to certain patients having allogeneic HSC transplants.
Results of the trial showed the safety of MultiStem therapy in this indication and suggested that MultiStem may have a beneficial effect in reducing the incidence and severity of GvHD, as well as providing other benefits.
At present, the company is preparing for a Phase II/III clinical trial in the area and expects to start th trial in 2014, depending on the progress in other clinical trials and the achievement of certain business development and financial objectives.