Aethersys Gets Orphan Status in EU for MultiStem

The EMA’s Committee for Orphan Medicinal Products has issued a positive opinion for Athersys‘ allogeneic, multipotent adult progenitor cell, or MultiStem therapy, for the prevention of graft-versus-host disease (GvHD). The company is developing its MultiStem cell therapy as a GvHD prophylaxis in patients undergoing allogeneic hematopoietic stem cell (HSC) transplant. The MultiStem therapy for the prevention of GvHD previously received Orphan Drug Designation from the FDA

Orphan drug designation, which is intended to facilitate drug development, provides substantial potential incentives to the sponsor, such as fee reductions for agency meetings, study-design assistance, opportunities for expedited product development, orphan grant access, tax incentives and market exclusivity for the product upon regulatory approval (seven years in the U.S. and as many as 10 years in the EU).

Patients with leukemia or other related malignancies are typically 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. These procedures are followed by a HSC transplant to reconstitute the immune system to fight infection and any remaining malignancy. Patients undergoing donor derived, or allogeneic, HSC transplants are at significant risk for serious complications, including GvHD, which results when transplanted immune cells attack various tissues and organs in the patient. GvHD can be severe and life-threatening, with substantial impact on overall treatment requirements and costs, as well as on the patient’s quality of life. Annually, there are estimated to be more than 25,000 allogeneic HSC transplants in the developed countries.

Athersys has completed a Phase I clinical study of the administration of MultiStem cells to certain patients having allogeneic HSC transplants. The study demonstrated 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. Aethersys is preparing a Phase II/III clinical study in the area. It has met with the FDA and received feedback regarding proposed plans for the next phase of clinical development and is finalizing the study design. Based on current plans, the company will be ready to start this study in 2014, but the initiation will depend on the progress in other clinical trials and the achievement of certain business development and financial objectives.