the opposition , good to know

Athersys' MultiStem(R) Stem Cell Therapy Receives Orphan Drug Designation in Europe for Prevention of Graft-Versus-Host Disease .EmailPrinter
FriendlyShare: facebook ? More.
.
StumbleUponLinkedInTwitterFarkRedditdel.icio.usMySpace Text Athersys' MultiStem(R) Stem Cell Therapy Receives Orphan Drug Designation in Europe for Prevention of Graft-Versus-Host Disease

European Orphan Designation Complements U.S. Orphan Designation for MultiStem Cell Therapy for Treatment of Patients with Leukemia or Related Malignancies Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

CLEVELAND, Dec. 17, 2013 (GLOBE NEWSWIRE) -- Athersys, Inc. (Nasdaq:ATHX) announced today that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has issued a positive opinion (EMA/OD/146/13) for the Company's allogeneic, multipotent adult progenitor cell, or MultiStem(R) 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 and is currently preparing for a Phase II/III clinical study in the area. The MultiStem therapy for the prevention of GvHD has also previously been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

"We are pleased to have been granted the benefits of orphan drug designation in Europe," said Dr. Manal Morsy, Head of Global Regulatory Affairs at Athersys. "Together with our U.S. 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."

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. These results build on the work of Athersys scientists and collaborators who have demonstrated that MultiStem cells suppress undesired T-cell-mediated immune responses that are an important factor in causing GvHD and support tissue repair and regeneration, leading to a significant increase in survival in preclinical models.

Currently, the Company is preparing for 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 is preparing to 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.

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 (7 years for the U.S. and up to 10 years for the EU).

Vinn