AASTROM'S CELL THERAPY GRANTED ORPHAN DRUG DESIGNATION

Aastrom Biosciences announced that its proprietary Tissue Repair Cells (TRCs) have been granted orphan drug designation by the FDA for use in the treatment of dilated cardiomyopathy (DCM).

Orphan drug designation is granted to novel therapeutics that offer potential value in the treatment of rare diseases and medical conditions, according to Aastrom. The company may be entitled to an expedited FDA review, the reduction or elimination of filing fees and possible tax credits.

DCM is a chronic cardiac disease that leads to enlargement of the heart and reduces pump function to a point that normal blood circulation cannot be maintained. Typically, patients with DCM show symptoms of congestive heart failure, including limitations in their physical activity and shortness of breath. DCM often represents the end stage of chronic ischemic heart disease in patients who have experienced multiple heart attacks. Other than heart transplant, there are no effective long-term treatment options for end-stage patients with this disease, according to Aastrom.

Scientific and early clinical evidence suggest that high doses of stem and progenitor cells may possibly slow down or reverse disease progression in the hearts of DCM patients, Aastrom said. TRCs, which include large numbers of stem and progenitor cells derived from a small sample of patients' own bone marrow, are intended to be used as a therapeutic to induce heart tissue regeneration. If successful, TRC treatment may eliminate or delay the need for a heart transplant, according to the company.